“We’re at an exciting juncture in medicine where the perceived scientific boundaries in treatment across therapeutic areas continue to dissolve.” These words from Jeffrey Jones, chief medical officer of Cullinan Therapeutics, encapsulated the general message from many of the 140+ biopharma leaders who told Scrip about their expectations for therapeutic area advances over the coming year.
“In 2025, I expect we’ll see further validation of therapeutic targets originally successful in one set of conditions applied to new diseases,” said Jones. “We’ve already started to see approaches like T-cell redirecting therapies, initially designed for cancer, reapplied to rein in overactive immune responses in autoimmune diseases. Leaning into these overlaps will enable the industry to focus less on rigid therapeutic categories and more on how to adapt targets and modalities to the biology at hand.”
Indeed, it becomes increasingly difficult each year to assign the predictions Scrip gathers to neat therapy area buckets, as the growing understanding of the complex interplay of biological mechanisms leads to reappraisal of diseases and the focus shifts from definition by symptom to definition by etiology. Nevertheless, the ultimate goal remains the same: to eliminate or at least alleviate patients’ symptoms, thereby improving their quality of life.
In 2025, “the biopharma industry faces the challenge of transforming novel advanced therapeutic technologies into market-ready treatments that are widely accessible to patients,” noted Monika Paule, CEO of gene editing company Caszyme.
Paule emphasized the need for smart, collaborative efforts to achieve this: “While progress continues, the journey from discovery to delivery remains complex and resource-intensive, particularly in an environment of constrained funding. These challenges are too significant for any single organization to address alone. Success requires a collective effort, uniting biotech and pharmaceutical companies around shared goals – advancing healthcare solutions and improving patient access. The year ahead demands a shift from ambition to collaboration, ensuring that breakthroughs provide meaningful benefits to those who need them most.”
Jazz Pharmaceuticals’ chief commercial officer Sam Pearce offered a concise summary of the therapeutic categories receiving the most attention.
“2024 has seemed like a year of change in the biopharma industry, with the huge growth of the GLP-1 category and the resurgence of the metabolic therapeutic area more generally,” she said. “However, if we dig into the numbers we can see that oncology remained the key growth driver for our industry and I expect this to continue into 2025, with other areas like neuroscience also continuing with growth trajectories seen in recent years.
“2025 will see a continued focus on previously un- or under-treated diseases and cancers, as through this focus we will be able to ensure that as an industry we are delivering meaningful value for patients.”
Oncology
Continuing advances in oncology therapy were highlighted by many of our commentators.
“The biopharma industry is poised for significant advancements in key areas of cancer, with particular promise in drugs with new mechanisms of action that better target and penetrate solid tumors while modifying the immunosuppressive nature of the tumor microenvironment,” said David Mazzo, CEO of Lisata Therapeutics. “The advancement of such drugs is expected to lead to marked improvement in treatment outcomes with the anticipation of a more tolerable adverse event profile for the patient. This new approach should maximize the benefit of combined therapies including chemotherapies and immunotherapies against these very difficult to treat tumors, ultimately paving the way to a new paradigm of treatment for solid tumors.”
“As we enter 2025, the landscape of solid tumor immunotherapy is undergoing a significant transformation,” agreed Tatiana Novobrantseva, chief scientific officer of NextPoint Therapeutics. “By combining immune system activation with direct tumor targeting, novel therapies like T-cell engagers and antibody-drug conjugates are emerging as promising treatments.”
She highlighted one field specifically. “A particularly exciting area of research is the B7-H7 axis. This novel checkpoint pathway, independent of PD-1/PD-L1, also represents a promising target for both T-cell engagers and antibody-drug conjugates. By targeting B7-H7, we may be able to overcome the limitations of existing immunotherapies and provide effective treatments for tumors that have developed resistance to current therapies.
“2025 promises to be a year of significant advancement in the field of immunotherapy,” predicted Novobrantseva. “We anticipate that classical and engineered with additional activity biologics will play a pivotal role in creating more intelligent and effective therapeutic approaches, ultimately improving patient outcomes.”
A particularly exciting area of research is the B7-H7 axis. This novel checkpoint pathway, independent of PD-1/PD-L1, also represents a promising target for both T cell engagers and antibody-drug conjugates.
Tatiana Novobrantseva, NextPoint Therapeutics
CatalYm’s chief medical officer, Eugen Leo, also expressed optimism around overcoming treatment resistance. “While cancer immunotherapy has made remarkable advances, resistance remains a major challenge, affecting the majority of solid tumor patients treated,” he said. “Targeting the immunologic pathways that allow cancer cells to evade immune detection and create an immunosuppressive microenvironment remains a critical industry focus in 2025. GDF-15, a locally acting immunosuppressive cytokine originally linked to stress responses, has emerged as a promising target. Over the past year, GDF-15 inhibitors have gained visibility with clinical data showing their ability to reverse resistance to checkpoint inhibitors in advanced solid tumors. They also hold potential in mitigating cancer cachexia, a debilitating condition characterized by significant weight and muscle wasting, which can significantly impact a patient’s quality of life and treatment tolerability. By addressing both immune resistance and cachexia, neutralizing GDF-15 has the potential to significantly improve patient outcomes and reduce treatment-limiting side effects. Advanced clinical trials will further explore this potential in the years ahead.”
“Cytokines will usher in I-O [immuno-oncology] 2.0,” declared Caroline Loew, CEO of Mural Oncology. “For the past decade, immuno-oncology has been defined by the blockbuster breakthrough PD-1 inhibitor class. A flurry of development, the nearing patent cliffs, and new combination agents on the horizon indicate promise for a second I-O revolution, driven by innovative approaches in cytokines, multispecific antibodies and T-cell engagers.”
“I believe three classes of anticancer medicines will make still bigger strides: ADCs [antibody-drug conjugates], bispecifics and bifunctional antibodies, as they offer more tumor targeted therapy than traditional mainstays,” said David Epstein, CEO of Ottimo Pharma. “In short, efficacy will increase, therapeutic windows will widen and patients will stay on medicines longer thus living longer, better lives.
I believe three classes of anticancer medicines will make still bigger strides: ADCs, bispecifics and bifunctional antibodies, as they offer more tumor targeted therapy than traditional mainstays.
David Epstein, Ottimo Pharma
“In particular in 2025 and beyond we will start to see a new generation of medicines with the modalities listed above coming to the fore as big pharma seeks to find replacements for the older checkpoint inhibitors, single-acting mAbs [monoclonal antibodies] and older chemotherapies,” he went on. “For example, we believe that by blocking two molecular pathways with one medicine, such as PD-1 and VEGF, it should be possible to produce drugs that will replace existing immunotherapy medicines becoming in time the new cancer backbone therapy to which other drugs are added.”
Tahi Ahmadi, chief medical officer and head of experimental medicines at Genmab, was also among those who highlighted the promise of ADCs.
“In 2025, the biopharmaceutical industry is poised to progress new and promising science in oncology. Particularly in the realm of gynecologic cancers, where substantial research and treatment gaps persist, biotech is uniquely positioned to advance new targets and innovative strategies. One of the most promising areas of innovation is the development of antibody-drug conjugates (ADCs) with advanced linker technology, which connects cancer-fighting drugs to antibodies, ensuring they are delivered directly to cancer cells,” he said. “This targeted approach minimizes harm to healthy cells and reduces side effects, potentially making treatments more effective and tolerable for patients compared to conventional chemotherapy. Novel linkers can be transformative, as they potentially allow for higher potency of the therapy while maintaining safety. By continuing to invest in technologies like these, the industry can make significant progress in closing the care gap for patients.”
In the view of George Eliades, CEO of Mythic Therapeutics, “we will continue to get closer to achieving the promise of ADCs – significant anti-tumor activity without the side effects of chemotherapy. Several innovations will fuel this, including new, innovative antibody technology in a field focused almost exclusively on linkers and payloads, and the ability to target a wider variety of tumors, including those with lower target expression. I also anticipate a renewed focus on duration of therapy and survival, as some of the high response rates we have seen in the ADC field have not translated to the most critical patient benefit – living longer.”
“Antibody-drug conjugates continue to be practice-changing for oncology patients while gaining market traction,” said Melissa Paoloni, chief operating officer, OncoNano Medicine, Inc. “However, I foresee polymer drug conjugates (PDCs) emerging as a transformative technology that addresses ADCs' fundamental limitations. Unlike ADCs, which require specific cell surface proteins for binding and can cause systemic toxicities due to instability in circulation, PDCs offer universal tumor targeting through pH-sensitive drug release and micelle-protected payloads, enabling more precise treatment across multiple cancer types. This innovative approach could significantly expand therapeutic options by decoupling drug delivery from biomarker selection and improve drug pharmacokinetics, potentially providing novel oncological treatment strategies in the years ahead.”
I foresee polymer drug conjugates (PDCs) emerging as a transformative technology that addresses ADCs' fundamental limitations.
Melissa Paoloni, OncoNano Medicine, Inc
For Bill Coyle, principal (global head of biopharma) at consultancy ZS, “while personalized, targeted medicines in oncology and other severe diseases continue to amaze, I’m particularly interested in the continued development and eventual registration of immune-oncology therapies combined with so-called personalized mRNA cancer vaccines.”
Mai-Britt Zocca, CEO of IO Biotech, countered: “In 2025, we anticipate a pivotal shift in oncology with the rise of off-the-shelf therapeutic cancer vaccines. These therapies offer broader accessibility and faster deployment than patient-specific approaches, such as custom-made mRNA vaccines. They’ll be useful across diverse patient populations, and in combination therapy settings they have the potential to boost the efficacy of standard-of-care checkpoint inhibitors without increasing toxicity.”
Michael Bauer, CEO of QUANTRO Therapeutics, highlighted another emerging area of promise. “The pharmaceutical industry has long sought to overcome the challenge of targeting transcription factors (TFs), key regulators of gene expression driving many diseases,” he noted. “Recently we saw a $1.8bn deal between Roche and Flare to discover new small-molecule cancer treatments via previously undruggable transcription factor targets, which confirms industry interest in this field.
“TFs have been largely considered ‘undruggable’ due to complex protein-protein-DNA interactions, often involving intrinsically disordered proteins. This has left drug discovery in this field reliant on serendipity. Emerging approaches such as proteomic screening and structure-based design offer promise, but face significant limitations, especially regarding uncertain biological relevance or undefined TF structures. Our approach focuses on time-resolved functional transcriptomics. Using metabolic labelling of nascent mRNA, we can precisely measure the production of mRNA directly in living cells, enabling the precise tracking of transcription processes during disease progression.”
For Stacy Lindborg, president and chief executive officer of IMUNON, interleukin-12 is the one to watch. “While IL-12 is well recognized for its potent anti-tumor properties, therapies designed to modulate IL-12 have been associated with risk of systemic toxicity that limits their clinical applications. Now, innovative strategies designed to administer IL-12 directly to the tumor microenvironment including virus-based, non-viral, and cell-based delivery are showing significant promise in preclinical and clinical studies. This past year even saw the first IL-12 immunotherapy achieve a clinically effective response including benefits in PFS and OS in treatment of ovarian cancer, with plans to advance into a Phase III trial in 2025. Recent advancements of novel IL-12 delivery strategies, with funding support from institutions such as the NIH and Dana-Farber Cancer Institute, show clear potential to develop therapies that can deliver optimal efficacy benefit with more manageable risk profiles. With this new momentum, it seems possible that an IL-12 therapy with these advantages could reach the market within the next decade.”
Cell and gene therapy approaches are also expected to advance further in the field of oncology.
“Currently, there are 12 approved cell and gene therapies for cancer, including seven CAR-T cell therapies for various blood cancers and the first TIL [tumor-infiltrating lymphocyte] and TCR [T-cell receptor] therapies for solid tumors. In addition, around 590 active clinical trials are advancing these therapies,” said Barbara Lavery, chief program officer at the Alliance for Cancer Gene Therapy.
“Remarkably, this progress has come within just eight years of the first breakthrough approval. By 2025, the robust pipeline of cancer cell and gene therapies will continue driving early-phase clinical trials, offering hope to patients with aggressive tumors like glioblastoma, DIPG [diffuse intrinsic pontine glioma] and pancreatic cancer. Innovations such as direct CAR-T cell infusions into the brain and personalized mRNA vaccines are showing promise,” she said. “Decades of dedication by pioneers like Carl June, Crystal Mackall, Christine Brown, Marcela Maus and Nino Chiocca are transforming outcomes for cancers with historically poor survival rates, making curative therapies not only possible but imminent.”
By 2025, the robust pipeline of cancer cell and gene therapies will continue driving early-phase clinical trials, offering hope to patients with aggressive tumors like glioblastoma, DIPG and pancreatic cancer.
Barbara Lavery, Alliance for Cancer Gene Therapy
“In coming years patients will be closer to benefiting from access to innovative and effective cancer treatments, due to the growing body of research around mucosal-associated invariant T (MAIT) cells to treat solid tumors, a huge unmet need as existing CAR-T therapies are approved for hematologic cancers only,” said Yaky Yanay, CEO and President of Pluri, Inc. “An increasing number of studies show that activated MAIT cells display significant anti-tumor activity. Placental MAIT cells show greater promise as an allogeneic cure, due to the reduced chance of graft-versus-host disease. They also have the potential for better migration into the tumor site compared to conventional T-cells due to the high levels of chemokine receptors they express. This could make them effective cancer treatments. The benefits for patients once these treatments hit the market will be tremendous.”
For more forecasts on cell and gene therapies, see subsequent chapters.
Radiopharmaceuticals are another area of significant interest.
“Advances in radiopharmaceuticals have already significantly improved patient survival rates – and overcoming current limitations could bring a transformative change in cancer treatment,” commented Daniel Steiner, senior vice president, research & technology at Molecular Partners. “Radiopharmaceuticals remain hindered by the lack of targeting moieties that can selectively target cancer cells, without causing toxicity to critical functions such as the bone marrow and kidneys. This has confined progress to targets with well-defined, ligandable pockets.
“Developing repeatable and reliable targeting vectors could bring a major shift. Radio-DARPins can target a wide range of cell surface proteins and have been engineered to overcome these limitations, demonstrating reduced kidney absorption of our proteins and high tumor uptake of DARPins with high affinity and specificity to such novel targets. Radio-DARPins are highly differentiated and ideal vectors for radiopharmaceuticals with exquisite targeting capabilities, attractive biodistribution profiles and ease of manufacturing. While DARPins can be used for a range of isotopes, the combination with 212Pb seems ideal. A first Radio-DARPin program at Molecular Partners, targeting DLL3 and in partnership with Orano Med, exemplifies this approach and efforts are being expanded.”
“In 2025, the biopharma industry is poised to witness substantial advancements in radioligand imaging and therapy within oncology,” said David Bejker, CEO of Affibody. “Innovations in radiopharmaceutical production and delivery, including the increased use of novel radioisotopes and more precise targeting mechanisms, will significantly enhance the effectiveness and safety of treatments. The ability to visualize treatment through radioimaging and facilitate dose-finding through dosimetry, leveraging insights from established and successful radioligand therapies, is expected to gain increasing support within the oncology community.
Innovations in radiopharmaceutical production and delivery, including the increased use of novel radioisotopes and more precise targeting mechanisms, will significantly enhance the effectiveness and safety of treatments.
David Bejker, Affibody
“At Affibody, we are committed to contributing to these advancements by expanding radioligand therapy into indication areas beyond the current mainstream of prostate cancer and neuroendocrine tumors,” Bejker said. “Additionally, developing radioligand therapies in a tissue-agnostic manner, as has been established for some antibody-drug conjugates (ADCs), is another promising direction for the field.”
“Targeted systemic radiotherapy has the potential to treat patients with remarkable precision,” said Michael Rossi, CEO of Y-mAbs Therapeutics. “The promise to target malignant disease and preserve healthy tissue makes the therapy very well tolerated. This would allow for the treatment of primary disease as well as micro-metastatic disease to help patients live longer, healthier lives.”
Homing in on a particular cancer was Biljana Naumovic, president, US solid tumor, Johnson & Johnson.
“I predict the new bladder cancer therapeutics will shock the world as the greatest oncology advancement in 2025,” she said. “Unlike treatments for many other types of common cancers, certain kinds of bladder cancer treatments haven’t significantly changed in 40 years. Today, patients’ options are bleak including BCG, the current standard of care (modified form of the tuberculosis vaccine) that has to be held in the bladder and requires post-operative care. The other is often surgery, including drastic bladder removal which is a life-altering procedure. Most of the advances in this space are either too toxic and complicated, or without meaningful long-term efficacy, or both. Fortunately, there is hope in innovative treatments designed to have patients keep their natural bladders that are showing transformational clinical promise. These advancements will get patients in front of cancer, because at J&J we will never settle until bladder and other cancers are behind everyone.”
For Lillian Smyth, global development head, breast cancer at Eli Lilly, “a major theme that will continue to shape priorities in breast cancer treatment is improving the patient experience, including for the thousands of people living with estrogen-receptor-positive (ER+) disease. The emerging class of oral selective estrogen receptor degraders (SERD) offers patients an alternative to standard of care endocrine therapy that doesn’t require intramuscular injection or additional visits to the doctor’s office. Oral SERDs offer patients flexibility where current treatment options are very limited. The opportunity to expand treatment options for patients who’ve experienced progression or recurrence is driving significant effort to rethink the way we approach endocrine therapy in breast cancer and remove long-standing burdens for patients.”
Quyen Nguyen, CEO of Alume Biosciences, highlighted advances in precision surgery. “The field of oncology is undergoing a remarkable transformation thanks to drugs that enable fluorescence-guided surgery. In recent years, three FDA-approved tumor-targeted fluorescent molecules have become available, offering surgeons a powerful tool for precise tumor resection. By illuminating tumor margins, these molecules allow surgeons to more accurately remove cancerous cells while sparing healthy tissue, significantly improving surgical outcomes,” she said.
The opportunity to expand treatment options for patients who’ve experienced progression or recurrence is driving significant effort to rethink the way we approach endocrine therapy in breast cancer and remove long-standing burdens for patients.
Lillian Smyth, Eli Lilly
“Having dedicated much of my career to researching this field, I am confident that the adoption of fluorescent drug technology for surgery will continue to grow. For instance, our team is currently conducting clinical trials with a fluorescent molecule designed for nerve identification in head and neck surgery – a critical innovation that could enhance surgical precision and patient safety. This is just the beginning. There is immense potential for similar advancements in diverse surgical settings, such as hernia repairs, spinal surgery, orthopedic surgery, prostate surgery and beyond,” Nguyen concluded.
Others noted the potential for improvements in early intervention.
“In oncology, there is a shift towards greater emphasis on preventing and treating early-stage cancers, driven by advancements in early detection technologies and precision medicine,” said Elisabeth de los Pinos, CEO of Aura Biosciences. “By focusing on earlier intervention, companies can improve survival rates and reduce the long-term burden of advanced cancer care. Aura Biosciences recently expanded its lead candidate bel-sar into bladder cancer, our second cancer indication, to treat earlier-stage cancer. I predict more companies will focus on earlier-stage disease to address unmet needs across multiple indications and prioritize prevention, precision, and improved outcomes for patients worldwide.”
“Advancements in cancer treatment over the past few decades have been truly remarkable. However, research has shown that more younger people are being diagnosed with cancer every year. As we grapple with this concerning trend, it will be imperative to address some of the long-term impacts of the gold-standard chemotherapies that have saved so many lives,” said Jeffrey S. Hackman, CEO of Fennec Pharmaceuticals.
“Unintended effects of platinum-based chemotherapy, such as permanent hearing loss or peripheral neuropathy, can significantly impact a patient’s quality of life – especially when survivors are children or young adults who have their entire lives ahead of them. I believe we will be seeing a paradigm shift in how healthcare providers approach these treatment considerations – where we prioritize proactive screening and early intervention to manage quality of life like we do the disease itself. By doing so, we can help ensure that patients not only survive cancer but also thrive post-treatment."
Andres Sirulnik, senior vice president, translational and clinical sciences, hematology, at Regeneron, was boldly optimistic. “We shouldn’t be afraid to use the four-letter word: cure. With a deep understanding of underlying disease biology and an ever-expanding toolkit of strategies – from genetic medicines, cell therapy to bi-specifics – we can finally offer the opportunity to shift our thinking from treating patients with blood cancers or disorders until disease progression to treating to cure,” he said. “It starts with asking ourselves clinically meaningful questions – can we intervene sooner at precursor stages before it becomes a cancer? Can we address the root cause of disease at the genetic level? Is cure possible? The answers to these questions are leaning to ‘yes,’ which means we as medicine makers are finally able to fulfill real promises to patients.”
We shouldn’t be afraid to use the four-letter word: cure. It starts with asking ourselves clinically meaningful questions – can we intervene sooner at precursor stages before it becomes a cancer? Can we address the root cause of disease at the genetic level? Is cure possible? The answers to these questions are leaning to ‘yes.’
Andres Sirulnik, Regeneron
Immunology & Inflammation
“Roughly 300 million people around the world suffer from immune-mediated diseases, which include psoriasis, inflammatory bowel disease, rheumatoid arthritis, Sjögren’s disease, several rare maternal-fetal conditions, and a variety of rare conditions. Most do not pursue advanced treatments, and many patients stop these because of loss of response. The fact that less than 10% of patients are considered in remission is simply unacceptable,” said David Lee, global immunology therapeutic area head at Johnson & Johnson Innovative Medicine. “My hope and expectation are that we make progress to address these gaps in the year to come. We deepen our understanding of the immune system and the mechanisms that drive inflammatory disease every year. New pathways and avenues of research are fueling a surge of interest and innovation in this field. Remission is our mission, and I expect progress towards this goal for more patients in 2025.”
Lee was not alone in highlighting the significant unmet need in this space.
“This past year global biopharma companies and leading industry investors poured billions into companies working in immunology and inflammation. However, the need for new and non-immunosuppressive treatments for autoimmune diseases continues to grow,” noted Dannielle Appelhans, CEO of COUR Pharmaceuticals. “While patients do currently have treatment options available, most are burdensome and do not address the underlying causes of disease. In 2025, I expect to see continued investment in the clinical advancement of technologies that work to target the root cause of autoimmune diseases through novel modalities.”
Judy Chou, CEO of AltruBio, concurred. “I expect the immunology and inflammation (I&I) field to continue to experience significant growth, building on the momentum from 2024, which saw major financing rounds and large-scale M&A deals,” she said. “This growth is fueled by a heightened prevalence and awareness of the diseases. The industry is increasingly focused on therapies with new mechanisms, instead of continuing to develop ‘me-too’ or ‘me-better’ drugs, that may only show incremental clinical improvements. I believe this will be achieved by developing drugs that restore immune balance by treating immune disorders at their source. This approach may also offer potential applications across multiple disease areas. A versatile approach, together with combination and/or precision therapies will be key to addressing this heterogeneous patient populations and to break the efficacy ceiling of many of the I&I diseases.”
In 2025, I expect to see continued investment in the clinical advancement of technologies that work to target the root cause of autoimmune diseases through novel modalities.
Dannielle Appelhans, COUR Pharmaceuticals
“The field of I&I continues its renaissance with activity expected to accelerate next year,” said Niels Riedemann, CEO of InflaRx. “Biopharma innovators such as InflaRx are directing their efforts to harness new mechanisms of action in the hopes of bringing new drugs to underserved patients across a variety of inflammatory conditions. Over time, we expect this innovation to serve patients by providing better and more durable treatment options, and also help expand a multitude of I&I markets in immune-dermatology, nephrology, neurology and beyond.”
“In 2025, we can expect a significant interest from investors in companies focusing on the anti-inflammation category, particularly targeting conditions like arthritis and other diseases,” said DeepCure’s CEO Kfir Schreiber.
“As the role of inflammation in these diseases becomes clearer, there will be a growing emphasis on developing novel therapies that can precisely modulate the immune response with minimal side effects.
“With advancements in drug discovery technologies, such as AI-driven platforms and biomarker identification, we’ll see a shift towards more personalized and effective treatment options for patients suffering from arthritis and even rare diseases like Still’s disease. AI drug discovery will continue to enable the development of better therapies that address inflammation directly, improving patient outcomes and more effective management of conditions like arthritis and Still’s disease,” Schreiber predicted.
“2025 will see the development of innovative new treatment approaches in the immunology and inflammation (I&I) therapeutic area. Many of the diseases in this area, such as the severely debilitating chronic skin disease hidradenitis suppurativa (HS), are poorly understood and underdiagnosed. The real incidence is estimated to be much higher, primarily as patients do not seek medical attention because of their devastating symptoms. The lack of disease awareness, combined with insufficient treatment options, have led to critical gaps in disease management,” said Jorge Santos da Silva, CEO of MoonLake Immunotherapeutics. “A wave of innovation in the space is leading to new treatment options for patients, which in the HS market alone is expected to reach $15bn by 2035. Novel approaches, such as Nanobodies [under development by MoonLake] may provide additional benefits compared to conventional monoclonal antibodies including their smaller size which can better penetrate affected tissues. MoonLake is conducting several Phase III trials which could result in the world’s first blockbuster Nanobody.”
For Daniel Faga, CEO of AnaptysBio, “2025 is shaping up to be a transformative year for the treatment of autoimmune disease with medicines with new mechanisms of action poised to challenge conventional treatment paradigms. At Anaptys, we have multiple Phase IIb data readouts from novel antibodies targeting co-inhibitory receptors on activated immune cells. This includes ANB032, targeting the BTLA co-inhibitory receptor and rosnilimab, targeting the PD-1 co-inhibitory receptor for patients living with atopic dermatitis and rheumatoid arthritis, respectively. This wave of clinical evidence could demonstrate the potential of these novel therapeutics to break through established efficacy ceilings in these complex and heterogeneous diseases.”
“Bridging the gap between evidence generated for medicines in development and maximizing their real-world impact when used by patients will become a focus in 2025,” said Kaivan Khavandi, senior vice-president of respiratory and immunology R&D at GSK.
Bridging the gap between evidence generated for medicines in development and maximizing their real-world impact when used by patients will become a focus in 2025.
Kaivan Khavandi, GSK
“In severe asthma, effective and consistent control of the perpetuating inflammatory process is important if we want to prevent exacerbations, disease progression and achieve next-generation treatment goals. Pioneering work has allowed the development of long-acting therapies that target this underlying inflammation for longer periods of time. When rates of persistence to biologics are low, factors like compliance and persistence need to be addressed.
“Next year, I expect a leap forward with a potential regulatory approval of the first ultra long-acting biologic treatment for severe asthma. [GSK’s] depemokimab has been shown to provide sustained suppression of a key marker of inflammation, reducing the risk of exacerbations, with two injections per year,” Khavandi went on. “Such solutions will draw a straighter line between mechanistic promise, trial data, and full clinical potential.”
A number of executives underlined the opportunity for advances in oncology to find applications in autoimmune disease treatment.
“We expect to see advances in therapeutics that address autoimmunity, driven by multivalent strategies and accelerated by discoveries in T-cell regulation in treating cancer,” said Tiba Aynechi, general partner at Norwest Venture Partners.
“Autoimmunity will continue to be an exciting area, as the application of therapeutic modalities that have shown benefit in oncology are progressed through development, and approaches targeted underlying and differentiated biology are accelerated, both in biotech and pharma,” said Greywolf Therapeutics CEO Peter Joyce. “Specifically, on the application of the new modalities, these will start to bear fruit, notably understanding potential for improved efficacy with various bispecific cytokine targeted approaches or in CAR-T therapies, understanding the trifecta of safety, efficacy and durability.”
“There is a compelling opportunity to expand patient impact through expanding our understanding of medicines that target B cells,” said Narimon Honarpour, senior vice president, global development, at Amgen. “Repositioning existing therapeutics, including those that have been oncology focused, toward indications in autoimmunity will be a key trend in 2025. We are leveraging our expertise in B-cell biology, target validation, and life cycle management to evaluate our medicines with purpose and in the service of patients living with autoimmune diseases.”
Repositioning existing therapeutics, including those that have been oncology focused, toward indications in autoimmunity will be a key trend in 2025.
Narimon Honarpour, Amgen
“When talking about therapeutic developments, we believe that 2025 will bring marked progress in the field of T-regulatory (Treg) cellular treatments of autoimmune diseases,” said Piotr Trzonkowski, CEO of PolTREG. “These treatments are predicated on the fact that Treg cells act as a regulator of the immune system, preventing the effector cells from attacking the body’s own tissues rather than the pathogens they are meant to eradicate. Almost always, autoimmune diseases are characterized by a low count or impaired activity in Treg cells, which companies like ours are hoping to remedy through autologous or allogeneic therapies. The therapies can be polyclonal, using in other words the patient’s own cells with no modifications other than their ex vivo multiplication. But there are also promising therapies with the much-debated CARTreg and other engineered types of Treg cells.”
“The biopharma industry is regaining interest in novel biological targets for serious diseases with significant unmet needs,” observed Mark Gaffney, CEO of Calluna Pharma. “In the immunology & inflammation sector, much of the recent deal activity has focused on developing best-in-class therapies against clinically validated targets (eg, GSK’s $1bn acquisition of Aiolos to broaden its range of asthma medicines). This renewed focus in first-in-class approaches, appears to follow saturation of earlier best-in-class attempts, with biotech companies increasingly delivering comprehensive, preclinical, and Phase I packages; the bar has risen, and biotech companies have stepped up to meet it. At Calluna Pharma, we see this shift firsthand as we are developing novel mAbs against first-in-class targets for fibrotic and inflammatory diseases; the interest from pharma rapidly increased the second half of this year with our Phase I SAD/MAD data in-hand, which should continue into 2025.”
For Christian Antoni, chief medical officer of Montai Therapeutics, however, “2025 will see the surging interest in immunology broaden beyond novel modalities to include effective and broadly accessible small-molecule solutions.”
2025 will witness greater movement in unlocking chemistry that can target known autoimmune disease drivers by focusing on untapped, diverse chemistry and leveraging AI to precision match chemical-to-biological relationships.
Christian Antoni, Montai Therapeutics
“Immunology is one of the hottest areas of drug development interest today, but the continued pursuit of complex novel modalities, like advanced biologics and cell therapies, is hindered by complex supply chains, high costs and limited access to global populations,” he warned. “2025 will witness greater movement in unlocking chemistry that can target known autoimmune disease drivers by focusing on untapped, diverse chemistry and leveraging AI to precision match chemical-to-biological relationships. This will open the door to delivering accessible, oral small molecules to the global population suffering from these prevalent diseases.”
“It’s clear that we need to continue to make progress in defining the molecular causes of autoimmune disease, not just using clinical presentation to group together diseases which are highly likely to have heterogeneous disease mechanisms,” said Tom Lillie, chief medical officer of Greywolf Therapeutics.
“This can lead to targeted precision approaches that are going to be critical to deliver truly disease-modifying therapies in autoimmune disease. Current available therapies like immunosuppressants and immunomodulators have broad impacts on the immune system and, while effective, are still not addressing the underlying disease mechanism causing the inflammatory response. These broad immunosuppressive approaches can also lead to serious side effects for patients. As our understanding of both autoimmunity and immune tolerance mechanisms evolves, we support the view that there is the potential for highly targeted therapeutic interventions. I expect 2025 will see further advances in precision medicines that specifically target and block the T-cell response in autoimmune disease, delivering therapeutics that are effective, and more tolerable for patients.”
“In 2025, the biopharma industry will place an even greater emphasis on identifying molecular and cellular biomarkers that predict treatment response, especially in areas like rheumatoid arthritis, where patients often cycle through multiple therapies before finding one that works,” said Diana Abdueva, CEO of Aqtual. “These biomarkers will enable more precise selection of therapies to individual patients, improving response rates and minimizing unnecessary exposure to ineffective treatments. The need for such biomarkers is driven by increasing patient demand for personalized care and the growing integration of advanced technologies, which are transforming the understanding of immune system dynamics and therapeutic outcomes.”
Immunotherapy Beyond Cancer
“Immunotherapy will come full circle in 2025 as IL-2 [interleukin-2] – the first approved cancer immunotherapy – is poised for a resurgence,” declared Carlos Bañado, CEO of ARScience Bio. “A growing body of evidence supports the use of IL-2 as an adjuvant or companion immunotherapy, given its ability to improve CD8+ T-cell response and modulate NK cells. Low-dose IL-2 is currently tested as a combination therapy in clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis and frontotemporal dementia, solid and hematologic cancers, and autoimmune diseases like type 1 diabetes and lupus nephritis. Improvements to delivery, including slow-release and oral formulations, can improve efficacy and adherence, meaning IL-2 is well-positioned to break through next year.”
“2024 has seen significant advancements in the development of multifunctional therapeutic antibodies, enhancing their efficacy and versatility in treating complex diseases,” noted Stefano Gulla, chief scientific officer of Kling Biotherapeutics. “A key innovation is the engineering of bispecific and trispecific antibodies, which can simultaneously target multiple antigens or cell types, improving precision in cancer therapies and reducing off-target effects. Additionally, antibody-drug conjugates have evolved, integrating potent cytotoxic agents with targeting antibodies, enhancing delivery to cancer cells while sparing healthy tissue. 2025 is poised to continue this trend with more assets exploring immune-modulating antibodies that engage multiple targets of the patient’s immune system to boost therapeutic outcomes. These multifunctional formats promise to address challenges in oncology, autoimmunity, and infectious diseases more effectively.”
The last 10 years revealed the T cell as the central immunotherapy player. The next 10 years will be the age of innate immune cells, which are so important to control T cell functions naturally.
Jonathan Kagan, Corner Therapeutics, Harvard Medical School, Boston Children’s Hospital
“The last 10 years revealed the T-cell as the central immunotherapy player. The next 10 years will be the age of innate immune cells, which are so important to control T-cell functions naturally,” said Jonathan Kagan, scientific cofounder, board member and distinguished scientist at Corner Therapeutics, Marian R. Neutra, PhD professor of pediatrics at Harvard Medical School and director of basic research and Shwachman chair in gastroenterology at Boston Children’s Hospital. “In the next few years, breakthroughs in our understanding of the innate immune system will unveil a wave of next-generation vaccines that could eradicate many cancers and infectious diseases. We are rapidly moving towards a future in which patient-friendly, low-cost vaccines are possible, and can broaden the spectrum of diseases treatable with immunotherapy.”
However, Kagan warned: “Concerns over the new US administration’s potential stance on vaccines and pharmaceuticals more broadly mean it is now more important than ever to emphasize the rigorous scientific underpinning of vaccine development. Public trust in immunization programs and medicine is critical: in 2025, the scientific and medical community must double down on communicating the tangible benefits these innovations bring to global health and civilization more broadly.”
“I expect continued growth and investment in the allergic disease space, driven by the rising prevalence of conditions such as food allergies like eosinophilic esophagitis, asthma, dermatological conditions such as atopic dermatitis, and rhinitis, along with a growing unmet need for innovative, patient-friendly solutions,” said Woody Bryan, CEO of Revolo Biotherapeutics.
“Advances have been made in the area, including a shift toward biologic therapies, such as monoclonal antibodies, some of which have recently received approvals. However, innovation will remain focused on developing therapies that provide better efficacy with improved safety profiles, offering patient-friendly administration options, and avoiding the long-term immunosuppressive side effects seen with current therapies like steroids or JAK inhibitors. Moving forward, there will be an emphasis on therapies targeting novel upstream mechanisms to regulate the immune response before it becomes problematic, rather than addressing it after the response has occurred,” Bryan predicted.
Manuel Llobet, CEO of Allergy Therapeutics, thought that “2025 promises to be transformative for allergy immunotherapy as we see breakthrough technologies moving through clinical development.” He noted: “The field is advancing beyond traditional lengthy treatment courses to innovative short-course therapies that could fundamentally change patient care. Particularly exciting is the progress in food allergies, where novel approaches like virus-like particle (VLP) technology are showing promise in early trials for conditions like peanut allergy – which affects 2-3% of the population in developed nations. These advances could offer real hope to millions currently living with the constant anxiety of severe allergic reactions. The convergence of better understanding of immune mechanisms with innovative delivery platforms is opening up possibilities for more effective, convenient treatments that could potentially be curative rather than just managing symptoms.”
Particularly exciting is the progress in food allergies, where novel approaches like virus-like particle (VLP) technology are showing promise in early trials for conditions like peanut allergy – which affects 2-3% of the population in developed nations.
Manuel Llobet, Allergy Therapeutics
Obesity, Diabetes, Metabolic Diseases
“As a CEO, I am deeply excited by the advances we’re making in the obesity therapeutic area. Tackling obesity starts with recognizing it as a disease, which is key to improving the overall health and quality of life for millions of people,” said CinRx Pharma CEO Jon Isaacsohn. “This year, we’ve witnessed groundbreaking research and the emergence of innovative therapies. Looking ahead, I believe we have the potential to transform how obesity is treated through newer mechanisms and combination approaches, offering patients new hope and long-term solutions to this complex, global health challenge.”
“We’ve seen tremendous interest and progress in the treatment of obesity, with GLP-1s among the best-selling drugs in 2024 and projected sales expected to reach $150bn by 2030,” said Jason Gardner, CEO of Ampersand Biomedicines and CEO-partner of Flagship Pioneering. “However, less than 4% of eligible patients take these approved drugs and there remains a significant need for new medicines that have improved therapeutic indices. I am confident that the convergence of advanced biological insights and cutting-edge technology, including AI, will not only broaden the range of potential therapeutic targets, but also redefine what is possible in metabolic diseases and beyond.”
“I believe more companies will focus on understanding the mechanisms of action underlying obesity due to the growing recognition of its complexity and the need for targeted treatments,” said Alex Aravanis, CEO of Moonwalk Biosciences. “By unraveling the genetic and epigenetic factors involved in obesity, companies can develop more precise and personalized interventions. At Moonwalk, we’re exploring these mechanisms to identify novel targets and biomarkers that can drive the development of innovative therapies. As we continue to gain insights into the complex biology of obesity, I expect that more companies will follow suit, leveraging advanced technologies like epigenetics to accelerate the discovery of effective treatments. In 2025, understanding the precise molecular pathways will be critical for addressing the growing global health challenge of obesity and its associated comorbidities.”
As we enter the next era of obesity therapeutics, I believe there will be a greater focus on different mechanisms of action. Dual agonists, triple agonists, and combination therapies will take center stage, driving advancements that address challenges like lean mass preservation, improved tolerability and the overall patient experience.
Vipin Garg, Altimmune
“Over the past year, the healthcare industry is realizing obesity therapies have the opportunity to transform health beyond weight loss. Emerging data continue to highlight how these therapies directly address co-morbidities associated with obesity leading to improved cardiovascular health, lipid profiles and reductions in liver inflammation,” said Vipin Garg, CEO of Altimmune. “As we enter the next era of obesity therapeutics, I believe there will be a greater focus on different mechanisms of action. Dual agonists, triple agonists, and combination therapies will take center stage, driving advancements that address challenges like lean mass preservation, improved tolerability and the overall patient experience. By investing in these next-generation approaches, we can unlock new possibilities and create therapies that have a positive impact on patients.”
Steffen-Sebastian Bolz, chief scientific officer of Aphaia Pharma, similarly noted the expansion in new mechanisms, as well as the potential for more precision: “The obesity field is set for significant advancements in 2025, with numerous clinical trials investigating approaches beyond GLP-1 agonists. Innovation is shifting toward alternative and combination therapies to improve efficacy and minimize side effects,” he said. “I expect to see more emphasis on approaches that deliver steady and sustainable over rapid weight loss. We are also gaining an enhanced understanding of treatment responses across diverse patient groups, influenced by factors like comorbidities, genetics, gender, or ethnicity. This progress underscores the importance of patient stratification and tailored treatments to ensure better outcomes, and is driving efforts toward personalized and precision medicine. As drug interventions deliver more benefit, we will see a stronger collaboration among drug developers, regulators, policymakers, and payers to ensure equitable access for a disease affecting significant portions of the global population. Scalable drug manufacturing will also be crucial to meet demand and support broader availability of these innovations.”
Punit Dhillon, CEO of Skye Bioscience, agreed that “2025 promises to be a year of significant progress for innovative approaches to obesity that extend beyond the approved GLP-1 receptor agonists. These advances will reflect a growing emphasis on patient-centric solutions prioritizing safety, tolerability and long-term effectiveness.”
He went on to identify a particular target of interest: “Among these, CB1 inhibition is gaining interest as a potential pathway to address alternative metabolic drivers of obesity. Early data from non-GLP-1 mechanisms, including CB1 inhibitors, suggest promising efficacy and improved tolerability, and opportunities to deliver sustainable and durable weight loss while also preserving lean muscle mass. New such pharmacotherapies integrated long-term with lifestyle modifications will expand the ability to address the diverse needs of obesity as well as comorbidities such as diabetes, kidney and liver disease, sleep apnea, and cardiovascular disease. A multi-faceted approach to drive improved patient health will better enable drugmakers to meet the needs of the one in eight people living with obesity globally.”
John Tsai, executive partner at Syncona and CEO of Forcefield Therapeutics, also highlighted the wider range of disease linked to obesity. “Metabolic diseases have absorbed a lot of the limelight this year and I expect this trend to continue into 2025. Anti-obesity medications have taken the pharmaceutical world by storm and have changed the way that we think about weight loss in society,” he observed. “Investors have followed the trend and there will be a boom of innovation in the diseases associated with obesity, including cardiovascular disease, kidney disease, and MASH [metabolic dysfunction-associated steatohepatitis] to name a few. We will see significant advances in these areas in the years to come.”
There will be a boom of innovation in the diseases associated with obesity, including cardiovascular disease, kidney disease, and MASH to name a few.
John Tsai, Syncona, Forcefield Therapeutics
“Amid a steadily increasing worldwide prevalence rate of cardiometabolic diseases, we have seen remarkable advancements in the cardiometabolic landscape, namely the first FDA approval of a therapeutic for patients with MASH,” said Rohan Palekar, CEO of 89bio. “Although patients with MASH now have a therapeutic option, one treatment will not adequately support this ever-growing patient population, as each stage of MASH requires unique treatment targets, with the primary treatment goal continuing to be reversing liver fibrosis and preventing cirrhosis. In 2025, I predict the momentum this approval has garnered will continue to cascade into incredible advancements in therapeutic options for cardiometabolic diseases, with attention on liver-directed therapeutic options. I also envision progress within regulatory agencies in terms of the adoption of non-invasive tests for evaluating treatments and monitoring patients with various cardiometabolic diseases. I eagerly await these improvements that better support the unique treatment needs of this large and growing patient population.”
“The use of prescription weight loss medications has swept across the globe, compelling a wide range of industries to strategize for this significant transformation. As a consequence, we are witnessing the emergence of new needs and patient profiles that require swift understanding and support,” observed Gerry Muhle, head of global product strategy, Galderma. “At Galderma, we are proactively investing in research and education to address the unique aesthetic challenges faced by people on their weight loss journey, including the impact on their skin – which may become dry and dull, sagging or appearing gaunt – and changes in their facial structure and balance due to loss of fat, collagen and elastin. Our aesthetics portfolio is uniquely positioned to address these needs, and our primary focus is equipping healthcare professionals with the necessary guidance to support these new groups, ensuring our solutions are tailored to meet their evolving needs.”
Highlighting type 1 diabetes was David-Alexandre Gros, CEO of Eledon Pharmaceuticals. “2025 stands poised to be a breakthrough year in the search for functional cures for type 1 diabetes, thanks to promising developments in both stem cell-derived islets and immunosuppressant innovation. Recent advancements in engineering stem cells to function as insulin-producing islets have demonstrated significant promise in early clinical trials, effectively mimicking the body’s natural insulin regulation. Meanwhile, promising clinical data of novel immunosuppressants are yielding better outcomes for type 1 diabetes patients receiving islet cell transplants, minimizing the risk of transplant rejection and significantly improving graft functionality compared to the current standard of care.
2025 stands poised to be a breakthrough year in the search for functional cures for type 1 diabetes, thanks to promising developments in both stem cell-derived islets and immunosuppressant innovation.
David-Alexandre Gros, Eledon Pharmaceuticals
“The year ahead brings critical milestones, as several of these technologies get ready to advance to Phase III clinical trials. Notably, 2025 is also expected to yield the first results from approaches aimed at allowing islet cell transplantation without the need for chronic immunosuppression, potentially transforming the treatment landscape. Such progress offers new hope to millions by moving closer to a future where these technologies are available to the broader patient community and diabetes management is redefined, focusing on restoring natural glucose control and reducing the burden of disease,” said Gros.
“Advances in islet transplantation for type 1 diabetes will take a significant leap forward in 2025,” agreed Camillo Ricordi, director emeritus of the Diabetes Research Institute, professor and chief of the division of cellular transplantation. “Emerging technologies, including devices for islet encapsulation and targeted delivery of immunosuppressants, promise to enhance graft survival and functionality while reducing systemic side effects. Stem cell-derived islets, having shown promise in pilot clinical trials, are now progressing to FDA Phase III registration trials, offering a scalable and renewable source of insulin-producing cells. With ongoing developments in immunomodulation and bioengineering, these therapies are poised to redefine the standard of care for type 1 diabetes, bringing us closer to achieving durable insulin independence for patients.”
Cardiovascular
“We are witnessing heightened interest from industry leaders, investors, and other key stakeholders in the cardiovascular therapy space,” said Sten Sorensen, CEO of Cereno. “One area to watch is the rising number of companies focusing on disease-modifying drugs, with several clinical readouts anticipated in the coming years. These innovations have the potential to address the growing patient demand for therapies that not only extend lifespan but also significantly enhance quality of life.”
“With cardiovascular disease-driven mortality rates increasing globally, 2025 promises to be a year of significant advancement in cardiovascular disease treatment,” commented Jon Congleton, CEO of Mineralys Therapeutics. “The prevalence of obesity and associated cardio-renal-metabolic (CRM) risk factors, such as dysregulated aldosterone and insulin resistance, is rising in tandem and driving a renewed focus on the future treatment of CRM conditions as a whole.
“Specifically, I anticipate a deeper understanding of the bidirectional relationship between obesity and related conditions, such as hypertension, chronic kidney disease and obstructive sleep apnea. Insights garnered by the increased research into these conditions, and their interconnectivity, will facilitate research and development of targeted therapies that stand to impact morbidity and mortality.”
“The global burden of cardiovascular disease has led to an increasing focus on drug development within the cardiometabolic disease space,” said Sandeep Kulkarni, CEO, of Tourmaline Bio. “With advancements in non-invasive imaging technologies, such as AI-powered cardiac imaging, ultrasounds, echocardiographies, computed tomography (CT) angiography, and nuclear imaging techniques, we may see these tools play an expanded role in refining trial designs and accelerating the evaluation of therapies for cardiovascular disease. Imaging has the potential to provide earlier and more precise insights into cardiovascular risk, enabling more efficient identification of high-risk patients and better tailoring of treatments. This shift could help strike a critical balance – leveraging advanced imaging to deliver innovative therapies to those in need while maintaining rigorous validation standards. By integrating cutting-edge technologies, we may redefine how cardiovascular disease is studied and addressed, with the ultimate goal of reducing the staggering rates of morbidity and mortality worldwide.”
“Cardiac precision medicine will remain a significant and growing opportunity in 2025,” commented Nolan Townsend, CEO of Lexeo Therapeutics. “As more targeted therapies enter late-stage trials and commercial markets, physicians and cardiac organizations are continuing to advance recommendations to shift to a more personalized approach in treating heart disease. There are several promising modalities under investigation including gene therapy, gene editing, RNA medicines and cell therapies.
I anticipate this ongoing innovation will be accompanied by an increase in genetic testing and further development of comprehensive cardiac screening panels, especially given the unmet need for genetically mediated cardiac diseases. These evolving trends should fuel further R&D and investment, which we saw accelerating already in 2024.”
As more targeted therapies enter late-stage trials and commercial markets, physicians and cardiac organizations are continuing to advance recommendations to shift to a more personalized approach in treating heart disease.
Nolan Townsend, Lexeo Therapeutics
Michael Jaff, vice president and chief medical officer, peripheral interventions, at Boston Scientific, also homed in on the cardiovascular space. “In 2025, the medtech and biopharma industry will increasingly prioritize developing therapies and technologies tailored to individual patient needs. Advances in data science and real-world evidence will play a pivotal role in this shift, allowing clinicians to leverage large datasets to fine-tune treatment strategies. This approach will ensure therapies are more precise and better aligned with each patient’s unique characteristics – ultimately improving outcomes and enhancing the patient experience with targeted, effective care,” he noted. “In the peripheral vascular space, there is growing excitement around the potential to develop more customized solutions for patients, such as in carotid interventions, where the benefits of Transcarotid Artery Revascularization (TCAR) continue to show outstanding clinical results across broad patient populations.”
Neurology
The field of neurology is undergoing something of a renaissance, with momentum building in a range of therapeutic areas, including neurodegenerative diseases like Alzheimer’s disease and Parkinson’s disease, psychiatry and multiple sclerosis. The interest is driven by opportunities for both improved diagnosis and novel treatment approaches.
“In 2025, I expect the biopharma industry to build on the strong momentum of recently approved treatments for Alzheimer’s by prioritizing the development of new Alzheimer’s therapies with improved safety profiles, particularly lower ARIA [amyloid-related imaging abnormalities, a side-effect of amyloid-beta therapies] rates,” predicted Daniel O’Connell, CEO of Acumen Pharmaceuticals.
“By focusing on novel approaches that enhance safety and broaden accessibility, there is potential to reach more patients and significantly improve their quality of life. Additionally, preferentially targeting other components of the Alzheimer’s disease pathway, such as toxic amyloid beta oligomers, offers an opportunity for next-generation therapies that can be used independently or in combination, providing greater efficacy and differentiated treatment outcomes. This progress of further innovation ensures that more patients benefit from treatments that could greatly enhance their quality of life,” O’Connell added.
“I anticipate a significant focus on novel therapies that go beyond antibody-based approaches. As current antibody therapies have shown limitations, particularly with brain inflammation, the focus will shift towards innovative solutions that offer both efficacy and safety,” said Christian Howell, CEO of Cognito Therapeutics.
“Inflammation-related side effects have prompted a shift towards innovative mechanisms, such as small molecules, genetic therapies, and medical devices and other disease-modifying solutions to address Alzheimer’s disease. This will likely include advancements in biomarkers and early detection technologies that facilitate timely intervention,” he continued.
“We anticipate a rise in therapies that utilize biomarkers for early detection and intervention, allowing for timely treatment before significant brain damage occurs. Additionally, there will be a push for non-inflammatory strategies that improve cognitive function and slow disease progression, reflecting a broader trend towards more patient-centric and personalized Alzheimer’s care.
“2025 will be a critical year in Alzheimer’s research, with the goal of developing safer, more effective treatments that improve quality of life and reduce the burden on patients and caregivers,” concluded Howell.
We anticipate a rise in therapies that utilize biomarkers for early detection and intervention, allowing for timely treatment before significant brain damage occurs. Additionally, there will be a push for non-inflammatory strategies that improve cognitive function and slow disease progression, reflecting a broader trend towards more patient-centric and personalized Alzheimer’s care.
Christian Howell, Cognito Therapeutics
“Our understanding of neurodegenerative diseases is advancing rapidly,” said Andrea Pfeifer, CEO of AC Immune. “We hope that within a few years we might be able to treat and even prevent these devastating conditions, which place a huge burden on patients, families, society and healthcare systems. Recent clinical data show that intervening early using anti-Abeta immunotherapy and anti-Tau-targeting approaches can improve treatment outcomes. This suggests that prevention using immunotherapy may be the best strategy for neurodegeneration – stopping the underlying pathology from progressing before neurons are lost and too many co-pathologies have kicked in. Active immunotherapies that use the person’s own immune system to slow onset and ultimately prevent neurodegenerative diseases could change the treatment paradigm. Targeting neuroinflammation to prevent the vicious circle of Abeta and Tau aggregation is another promising angle, which could complement immunotherapeutic approaches. And overall, I expect combination therapies will become an increasingly important strategy to tackle the multifaceted causes of neurodegeneration.”
“Parkinson’s disease and related neurodegenerative disorders affect millions, but over 30% of patients are misdiagnosed particularly earlier in the disease process. Recent scientific innovation has paved the way for biomarker tests that can detect the presence of abnormal proteins in symptomatic patients before these diseases progress to advanced stages,” commented Richard Morello, CEO of CND Life Sciences. “Clinicians and patients are now benefiting from the use of these biomarker tests in everyday practice, while pharmaceutical companies incorporate them into trial designs for drugs intended to modify the underlying causes of these debilitating diseases. For studies focused on Parkinson’s disease, dementia with Lewy bodies, and related disorders, identifying and quantifying the alpha-synuclein biomarker in cutaneous nerves has shown real promise as a tool for patient enrollment criteria and target engagement assessment and may play a long-term role in the development of novel therapies that are so desperately needed.”
“With advancements in technology, including the application of AI, and the broadening of treatment modality options, the year ahead holds great promise for major progress in several therapeutic areas, including neurodegeneration, where we expect to see significant developments in both central nervous system and retinal diseases,” said Alexander Gebauer executive chairman of Galimedix. “Glaucoma, for example, long viewed primarily as an intraocular pressure issue, is increasingly recognized as a complex retinal disease, opening doors for innovative approaches.
“In Alzheimer’s disease, 2024 saw the approval of another antibody; looking forward, alternative modalities, including small molecules, are being explored and hold the promise of overcoming many of the challenges with existing products. I am excited about the promise AI holds to truly reshape drug development, from more precise trial designs to potentially replacing placebo arms with AI-based disease progression modeling. All this not only accelerates development but also brings us closer to more personalized and effective treatments.”
Kelly Abernathy, vice president of clinical development at Arrivo BioVentures, outlined “five transformative shifts” that the “neuropsychiatric clinical trial landscape is undergoing […] in 2025.”
She said: “First, treatments are moving beyond traditional receptor-based approaches to target ‘upstream’ mechanisms that repair rather than mask symptoms.
“Second, the use of AI and real-world data analytics are revolutionizing trial design, resulting in reduced costs and accelerated timelines.
“Third, there’s growing recognition of sex-specific disease mechanisms, with researchers finally addressing sex differences, particularly in conditions twice as common in women, like depression and Alzheimer’s.
“Fourth, decentralized clinical trials (DCTs) are becoming more common, facilitated by recent regulatory guidance and growth of DCT service providers, making participation in clinical trials more accessible and enhancing diversity.
“Finally, subjective symptom rating scales administered during weekly clinic visits are being replaced by real-time, wearable digital technologies, imaging and biomarkers, providing scientifically validated, objective measures of safety and efficacy.”
For Abernathy, “these convergent trends are ushering in an era of more precise, personalized neuropsychiatric medicine.”
“The substantial unmet need in neurology comes with daunting drug development challenges, and the space has been littered with failures,” acknowledged Kirk Brown, Alnylam Pharmaceuticals’ vice president of research, who nevertheless saw reason for optimism. “Nucleic acid therapeutic approaches, such as RNA interference (RNAi), work upstream of conventional medicines to target the root cause of diseases. RNAi therapeutics can silence any gene in the genome, so long as they get to the right cells. On the heels of RNAi’s success in the liver, technological advances have unlocked additional tissues, including a novel C16 conjugate that delivers RNAi therapeutics to the brain,” he noted.
As scientists continue to enhance delivery features and conquer blood-brain barrier crossing, we will potentially see RNAi therapeutics impact a broad range of neurological diseases.
Kirk Brown, Alnylam Pharmaceuticals
“There is a palpable sense of hope for neurology drug development in 2025. RNAi therapeutics are now being investigated for Alzheimer’s disease, Huntington’s disease, and other devastating neurological conditions,” said Brown. “We’re cresting the next wave in nucleic acid therapeutics. As scientists continue to enhance delivery features and conquer blood-brain barrier crossing, we will potentially see RNAi therapeutics impact a broad range of neurological diseases.”
“A new era of neurotherapeutics is arriving at a critical time for society,” said Meg Alexander, chief operating officer of Ovid Therapeutics. “Gentler medicines with novel mechanisms of action (MOAs) are needed, particularly as the baby boomer generation ages into a ‘silver wave.’ Improved longevity will increase the familial and societal burden of dementias, Parkinson’s disease, and seizures. To address these and other intractable brain disorders, medicines developers are seeking to drug fundamental biological targets that underly disease pathophysiology. Excitingly, neurology is making significant progress. For the first time in 20 years, a new MOA was approved for schizophrenia and huge progress is being made with innovative therapeutic approaches to quell hyperexcited neurons across several therapeutic areas, from epilepsy to pain and psychiatric disorders.”
Kees Been, CEO of Kynexis, also highlighted the progress in schizophrenia. “One of the most anticipated regulatory decisions of 2024 was the approval of BMS’s novel schizophrenia drug, marking the first new treatment for the condition in decades,” he said. “This breakthrough has reignited excitement in the field as we continue to deepen our understanding of the disease and its impact on patients. Cognitive impairment, which affects the majority of those with schizophrenia, remains a critical challenge in delivering a meaningful treatment for the disease. With growing insights into the underlying mechanisms driving cognitive impairment in schizophrenia, coupled with innovative approaches in precision medicine, we are entering a new therapeutic era that holds great potential for transforming the lives of schizophrenia patients.”
Terran Biosciences’ CEO Sam Clark noted that “throughout 2024, there’s been a significant increase in investment and M&A in the neuropsychiatric treatment space, specifically around new classes of compounds emerging to further improve the patient treatment paradigm for diseases previously thought to be untreatable.
“With Bristol Myers Squibb’s Cobenfy becoming the first FDA-approved drug to treat schizophrenia targeting muscarinic receptors, it has opened the door for other companies to advance treatments with a similar mechanism of action in 2025,” Clark said. “For example, a novel prodrug approach could leverage Cobenfy’s approval to accelerate the development pathway to further improve the standard of care, reduce side effects, and optimize dosing schedules for a better patient experience. We expect the innovations to continue, with new mechanisms and therapeutic candidates emerging in schizophrenia and across the entire neuropsychiatric space.”
With Bristol Myers Squibb’s Cobenfy becoming the first FDA-approved drug to treat schizophrenia targeting muscarinic receptors, it has opened the door for other companies to advance treatments with a similar mechanism of action in 2025.
Sam Clark, Terran Biosciences
“Given significant unmet need, and promising early results, the industry will continue to focus on developing innovative treatments for neuropsychiatric conditions, such as major depressive disorder and more, which address the root causes of these diseases by repairing and restoring damaged neurons,” said Mark Rus, CEO of Delix Therapeutics. “Neuroplastogens, which are engineered to physically heal the brain through a process called neuroplasticity, but without the harmful side effects associated with first- and second-generation psychedelics including hallucinations and cardiotoxicity, have the potential to address some major neuropsychiatric conditions in new and innovative ways. I look forward to seeing the impact neuroplastogens may have in the next generation of neurological treatments.”
Daniel Karlin, chief medical officer of MindMed, thought that psychedelics would play an increasingly important role in the field. “Long dismissed from medicine and relegated to the fringes of society, psychedelics are re-emerging as potential treatments for brain health disorders. Now supported by rigorous studies, one dose of LSD and the resulting period of perceptual alteration has been demonstrated to yield benefits in patients with generalized anxiety that persist long after the transient effect of the drug subsides. This marks a transformative era in psychiatry, paralleling past medical shifts like the adoption of anesthesia which radically transformed surgery. These developments highlight the importance of integrating psychedelics within structured psychiatric frameworks to ensure safety and effectiveness, providing a hopeful path for millions affected by mental illness,” he said.
Sanofi’s head of R&D Houman Ashrafian, expected progress in the treatment of multiple sclerosis thanks to the company’s BTK inhibitor tolebrutinib, which was granted breakthrough therapy designation by the US FDA in December. “Tolebrutinib represents an unprecedented breakthrough in multiple sclerosis as a potential first-in-disease treatment option with clinically meaningful benefit in disability accumulation,” he said. “Addressing disability accumulation, thought to be driven by smoldering neuroinflammation, remains the greatest unmet medical need in people with non-relapsing secondary-progressive MS today. Tolebrutinib has demonstrated its ability to delay disability by targeting underlying drivers of the disease. To date, the primary target of current therapies has been peripheral B- and T-cells, while innate immunity, which is believed to drive disability accumulation, remains largely unaddressed by current therapies. Tolebrutinib’s mechanism of action modulates both B lymphocytes and activated microglia in the central nervous system, which is understood to address the underlying mechanisms of disability accumulation in MS linked to smoldering neuroinflammation in the brain and spinal cord. Regulatory submissions of tolebrutinib in nrSPMS are being finalized for the US and prepared for the EU.”
Beyond pharmaceutical interventions, Mike Kelly, CEO of NervGen, predicted that “we will see significant advancements in neuroscience in 2025, driven by new technologies, artificial intelligence, cutting-edge neuroimaging, and other emerging technologies such as brain-computer interfaces (BCIs) and neural implants. Many of these advancements can be combined with new therapeutic approaches and cell and gene therapies, which will broaden our understanding of brain function and neuro-regeneration pathways. It’s an exciting time and hope is building to bring some of these advancements to patients worldwide.”
Aging
“The biopharma industry is undergoing a transformative shift, driven by longevity science and the need to manage chronic diseases,” said David Bearss, CEO of Halia Therapeutics. “GLP-1 agonist drugs have sparked new thinking in addressing aging populations and declining birth rates, marking the beginning of a new pivot toward therapies that not only treat but also prevent chronic conditions, extending both lifespan and healthspan. As innovative treatments targeting the root causes of aging and chronic conditions approach clinical trials, we must adopt new development and regulatory frameworks to foster these breakthroughs.”
“The focus in biopharma has shifted from broad ‘aging investment’ to targeted therapies for specific diseases that impact aging, such as neurodegenerative, cardiovascular, metabolic, oncologic and inflammatory conditions,” observed Lola Buono, associate at Angelini Ventures. “In my opinion, addressing these diseases leveraging the latest technological innovations in the biotech space – such as gene therapies for neurodegeneration, RNA-based treatments for metabolic disorders, and targeted therapy approaches in oncology and drivers of the disease – are proving to be the effective way to improve quality of life rather than simply extending lifespan. What we see is a growing emphasis on interventions that tackle the root causes of age-related diseases, moving beyond the bare pursuit of longevity. A key trend we are witnessing is the increasing convergence of advanced biological research and translational medicine to accelerate the development of therapies that promote healthy aging. Rather than focusing solely on lifespan extension, the industry is recognizing that the most meaningful progress comes from treating the conditions that drive functional decline, ensuring that extended years are lived in good health.”
A key trend we are witnessing is the increasing convergence of advanced biological research and translational medicine to accelerate the development of therapies that promote healthy aging.
Lola Buono, Angelini Ventures
“In 2025, the emergence of senolytic therapies aimed at eliminating senescent cells is set to revolutionize our understanding of biological aging and cancer biology,” predicted Thomas Ichim, chief scientific officer of Immorta Bio. “Senescent cells play a pivotal role in the origin and progression of cancer and shield tumors from standard therapies. By leveraging targeted small molecules or advanced immunotherapies like SenoVax –our proprietary [preclinical] senolytic treatment – we can unlock the body’s natural regenerative capabilities, address the root causes of age-related diseases, and overcome mechanisms driving cancer resistance. This breakthrough promises to usher in a new era of extended healthspan and enhanced quality of life.”
Rare Diseases
“Rare diseases represent one of the most pressing challenges in medicine today. With up to 10,000 known conditions affecting nearly half a billion people globally these diseases are individually rare but collectively common. They are often fatal and frequently affect children, yet there is no effective treatment for 95% of patients,” said Matthew Wood, director and chief scientific officer of Oxford-Harrington Rare Disease Centre.
“Genetic medicines are already proving transformative for treating rare genetic diseases and in 2025 we expect to see further progress in this area,” Wood went on. “Technological advances and approaches, such as ‘therapeutic genomics,’ have opened opportunities to scale the development of genetic therapies across multiple diseases in parallel. By leveraging proven delivery platforms to accommodate different corrective genetic components, we can remove the need to restart the development process each time, saving time and cost while expanding access.
Technological advances and approaches, such as ‘therapeutic genomics,’ have opened opportunities to scale the development of genetic therapies across multiple diseases in parallel.
Matthew Wood, Oxford-Harrington Rare Disease Centre
“A highly supportive ecosystem for rare diseases is also emerging and we expect to see increased collaboration, not just between researchers and patients, but also with regulatory bodies, the biopharma industry and investors, to drive the development and availability of these urgently needed treatments for patients and their families.”
“2024 was a transformative year for research and development advances in rare diseases across multiple modalities, including RNA therapeutics, gene therapies, and small molecules,” said Sarah Boyce, CEO of Avidity Biosciences. Highlighting Avidity’s work with antibody oligonucleotide conjugates (AOCs), designed to address the root causes of rare diseases with limited or no treatments, she noted: “This year, we presented groundbreaking clinical data for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD). These results showcased the consistency and reproducibility of our platform – from delivery to target engagement – leading to meaningful functional improvements. Looking ahead, we anticipate even greater momentum in therapeutic advancements across rare and common diseases as innovative platforms like ours redefine what’s possible. Collaboration across the industry will be key to accelerating progress and bringing life-changing treatments to those who need them most.”
Manuela Maronati, global asset head, neuroimmunology and rare diseases, at UCB, said: “In rare diseases, such as generalized myasthenia gravis (gMG), a growing understanding and appreciation of the individual experiences and needs of patients is bringing much-needed advances in the treatments available and I believe this will continue in 2025. For people living with myasthenia gravis their symptoms vary greatly, so every individual’s experience is different and this can lead to profound uncertainty. As such, the condition can have a considerable impact on people’s daily lives, and they may also need different choices when it comes to in-hospital or at home treatment administration. For these reasons, it is crucial to prioritize access to next-generation therapy options that may offer personalization according to people’s unique requirements and disease profile. In this way, by listening to and treating the individual rather than the disease we can aim to deliver better outcomes.”
As biopharma continues to innovate, the focus must remain on delivering solutions that not only address underlying disease mechanisms but also enhance patients’ quality of life.
Kent Hawryluk, MBX Biosciences
Kent Hawryluk, CEO of MBX Biosciences, had a similar take. “As biopharma continues to innovate, the focus must remain on delivering solutions that not only address underlying disease mechanisms but also enhance patients’ quality of life,” he said. “For chronic and rare conditions, like hypoparathyroidism, long-term therapies that are both effective and convenient can be transformative. Advances in peptide engineering, prodrug platforms and technologies to extend time action are paving the way for treatments that align with patients' needs – reducing the burden of frequent dosing while maintaining durable, consistent therapeutic benefits. By prioritizing these patient-centered advancements, we have an opportunity to redefine standards of care in underserved areas.”
“Industry trends often result in therapeutic areas and approaches coming in and out of favor based on factors that include clinical trial outcomes, investor interest and general innovation in the space,” commented Jeremy Springhorn, CEO of Nido Biosciences. “One such area that has ebbed and flowed over the years is neuroscience and one that is currently out of favor is rare diseases. I anticipate that as we progress through 2025 and beyond, we will see interest in rare disease drug development gain momentum. There is an expansive patient population who experience significant unmet need for therapeutic options, and I believe that investors and drug developers will take notice of this despite what some have viewed as unfavorable public policy outcomes effecting the space in recent years.”
Musculoskeletal
“I’m energized as I see advancements in nonsurgical interventions for orthopedics and musculoskeletal conditions that not only give patients alternatives to surgery but can also help them maintain the lifestyles they want,” said Patrick Barry, president, global commercial operations at Endo. “As the global population continues to age and strive to maintain an active lifestyle, it’s imperative that the biopharmaceutical industry advances scientific and medical innovations that will help people maintain mobility and a high quality of life. By investing in nonsurgical approaches, we can offer treatments to patients that can potentially help improve recovery time and improve overall healthcare costs, while maximizing patient outcomes. By addressing musculoskeletal challenges with nonsurgical solutions, the biopharmaceutical industry raises the bar for patient care and enables more people to lead the active lives they want well into their later years.”
By investing in nonsurgical approaches, we can offer treatments to patients that can potentially help improve recovery time and improve overall healthcare costs, while maximizing patient outcomes.
Patrick Barry, Endo
“I foresee a significant resurgence in skeletal muscle research within drug development, paving the way for potentially revolutionary treatments for both rare neuromuscular diseases and larger indications such as sarcopenia,” predicted Thomas Holm Pedersen, CEO of NMD Pharma. Skeletal muscles play pivotal and diverse roles in the human body, making up 40-50 % of the body weight in humans. In addition to enabling mobility, skeletal muscles play key roles for metabolism, endocrine signaling, and even the immune system. They represent an untapped potential for effective treatments that could significantly improve patient outcomes and quality of life. Breakthroughs in this field offer hope for addressing conditions such as rare neuromuscular diseases, age-related muscle loss, and metabolic diseases. It’s an incredibly exciting time, with the potential for transformative therapies that could change the landscape of medical treatment for these conditions.”
Infectious Diseases
“In 2025, the biopharma industry will continue to prioritize proactive strategies to address global health challenges, with a particular focus on infectious diseases,” predicted Derek Wallace, president of Takeda’s global vaccine business unit.
“The rise of mosquito-borne illnesses like dengue, malaria, and chikungunya –exacerbated by climate change – highlights the urgency of advancing vaccination efforts, as well as strengthening disease surveillance and public health systems. Preparedness against outbreaks requires not only innovation but also collaboration. A unified effort from health authorities, the private sector, and at-risk communities is essential for developing coordinated solutions. These measures are critical to managing current health threats and building resilience against future challenges,” Wallace said.
“In 2025, biodefense solutions will continue to grow as the global leaders recognize the urgent threats posed by infectious diseases,” said Diem Nguyen, CEO of SIGA Technologies. “Recent events have underscored the importance of preparing for outbreaks – whether natural, accidental, or intentional – and highlighted gaps in existing response strategies. There will be increased emphasis on developing scalable antiviral therapies, research into better detection methods, and robust stockpiling systems to safeguard public health. The year ahead could bring transformational strategies with primary objectives to enhance preparedness and strengthen biodefense frameworks worldwide.”
“R&D for self-amplifying RNA vaccines is set to soar in 2025 as the technology provides more persistent immune responses and, thus, protection compared to linear mRNA vaccines. The demand is timely given multiple recent threats of pandemic-level outbreaks (Mpox, avian flu), and saRNA has the potential to create more potent, effective vaccines – all vital qualities as world governments continue pandemic preparation and response efforts,” noted Kate Broderick, chief innovation officer of TriLink Biotechnologies.
The year ahead could bring transformational strategies with primary objectives to enhance preparedness and strengthen biodefense frameworks worldwide.
Diem Nguyen, SIGA Technologies
“As we look ahead, the need for new modalities to treat influenza becomes increasingly urgent,” said Jeffrey Stein, CEO of Cidara Therapeutics. “Each year, seasonal influenza continues to evolve, with strains often outpacing current vaccines and treatments. This can lead to reduced vaccine efficacy and challenges in managing outbreaks. Additionally, the global burden of flu-related hospitalizations and deaths, especially in immune-compromised populations, highlights the limitations of current antiviral drugs and preventative strategies. There is also growing concern about the potential for pandemic flu strains, such as H5N1, which could have far-reaching consequences. To address these challenges, it’s crucial to advance innovative therapies that target the virus more effectively, including broad-spectrum antivirals with universal activity across multiple influenza strains. Developing these new modalities will be essential in reducing flu’s impact, improving patient outcomes, and enhancing our ability to respond to future pandemics.”
It’s crucial to advance innovative therapies that target the flu virus more effectively, including broad-spectrum antivirals with universal activity across multiple influenza strains.
Jeffrey Stein, Cidara Therapeutics
Lawrence Blatt, CEO of Aligos Therapeutics, highlighted hepatitis B as a “landscape ripe for significant change.” He said: “After more than 25 years without a new mechanism of action and low functional cure rates, innovative therapies are finally emerging as hepatologist and virologists finally have a deeper understanding of HBV biology. As a result, I think 2025 and beyond will bring transformative treatment options that offer new hope for HBV patients.”
For James Graham, CEO of Recce Pharmaceuticals, “antimicrobial resistance is an ever-growing area of concern, with more bacteria becoming resistant to available therapeutics. Patients suffering from difficult-to-treat infections, such as acute bacterial skin and skin structure infections (ABSSSI), diabetic foot ulcers, or complicated urinary tract infections, may need multiple rounds of antibiotics to kill their infections or run the risk of developing sepsis. As we move into 2025, I anticipate raising awareness around superbugs and the impact they have on not only the health of the globe but also the significant financial burden they place upon healthcare systems. I expect significant investment into research and development of novel antimicrobial solutions that not only effectively kill superbugs but do not further contribute to antibiotic resistance.”
Rod Cotton, venture partner, 2Flo Ventures, homed in on sepsis as an area to watch this year. “For decades, high-level research has overlooked some of our most essential and impactful healthcare challenges. A key example, and an area of profound unmet need is sepsis and septic shock,” he commented. “Sepsis is a leading cause of death, accounting for approximately 6% of all US hospitalizations and 35% of all in-hospital deaths. An equal opportunity killer, sepsis presents an ever-greater challenge to lower socioeconomic populations. Thankfully, innovation is coming. Diagnostics powered by AI offer the opportunity to recognize sepsis in the earliest stages, allowing for a more rapid response. Even more exciting, new therapeutic treatments are under development to address the condition, which offer the promise to save lives. Some of these are in later stage development with the potential be available to hospitals and patients in the very near future.”
Infection management is not the first thought most have for pain control, but this is the approach being taken by Persica Pharmaceuticals. CEO Steve Ruston explained: “In 2025, I believe we will see significant advancements in pain management. We are particularly focused on chronic low back pain (cLBP), which affects about 35 million patients in the USA alone. Of these, 7-9 million individuals suffer from moderate to severe symptoms that are not helped by existing treatments, leaving people reliant on analgesics, such as opioids, to manage pain.
“Research has shown that bacterial infection in intervertebral discs linked to Modic changes in the vertebrae can be a key driver of persistent cLBP, and that high-dose oral antibiotics can deliver unprecedented pain reduction. However, prolonged antibiotic therapy poses challenges for patient compliance as well as antibiotic stewardship. At Persica, we are developing a novel antibiotic preparation for intradiscal injection to provide a potentially curative opioid-sparing treatment. With clinical data expected in Q1, our goal is to transform pain management for millions of people in need.”
Ophthalmology
“We’re seeing transformative growth in the ophthalmology treatment landscape, and I anticipate additional data that support successfully addressing the treatment burden for patients with chronic and debilitating retinal diseases in 2025,” predicted 4DMT’s chief development officer Dhaval Desai. “Current therapies are effective in slowing disease progression and, in some cases, preserving vision in patients. Yet, treatments on the market today require frequent clinic visits and are unsustainable over the long term for many patients. While incremental advancements have provided treatment extension, the need for more durable and longer-term solutions, that also preserve vision, remains. Emerging therapies, such as genetic medicines, hold the promise to energize the ophthalmic landscape by demonstrating robust safety and potential use long term – while preserving vision. As these treatments advance, they have the potential to reshape how we address ophthalmic diseases and provide more sustainable care models to patients in need.”
Anirvan Ghosh, CEO of Unity Biotechnology, concurred. “New therapeutic approaches in ophthalmology are poised to disrupt the treatment paradigm in retinal diseases, where anti-VEGFs have been the standard of care for over 15 years,” he said.
“Interest in the ophthalmology space has been driven by the emergence of drugs based on new mechanisms of action, clinical and commercial success, and major strategic transactions. From senolytic therapies to genetic medicines, the most transformative treatments will not only provide long-lasting benefit and disease stabilization, but effectively remodel the retina, promote revascularization of healthy retinal tissue, and change the course of these vision threatening diseases,” Ghosh said. “In 2025, we’ll start to see the culmination of these efforts as novel therapies and advanced technologies enter late-stage clinical development with the potential to disrupt the current treatment landscapes and provide a new class of therapeutic options for patients suffering from progressive vision loss. I predict we will have new standards of care for patients with chronic retinal diseases in the near future.”
I predict we will have new standards of care for patients with chronic retinal diseases in the near future.
Anirvan Ghosh, Unity Biotechnology
“The biopharma industry’s adoption of precision medicine approaches will likely extend beyond oncology in 2025 and in the years to come,” said Riad Sherif, CEO of Oculis. “While targeted therapies have become the standard in cancer treatments, other therapeutic areas such as ophthalmology are beginning to explore biomarker-driven patient selection. General ophthalmology, traditionally reliant on broad therapeutic approaches, is seeing first applications of precision medicine in clinical trials. This exciting advancement is representing a fundamental shift away from the trial-and-error approach which often leads to frustration for physicians and patients. As our understanding of genetic biomarkers improves, more therapeutic areas will likely begin incorporating precision medicine approaches, learning from oncology’s success in identifying patients most likely to respond to specific treatments and predict outcomes. This evolution could lead to more efficient clinical development pathways and better-targeted treatments that deliver improved results for patients, physicians and payers.”
Paul Bresge, CEO of Ray Therapeutics, highlighted the “growing need for novel therapies in retinitis pigmentosa (RP), particularly companies developing novel therapies focused on delaying vision loss, or potentially even restoring vision.”
Noting that “this rare and degenerative eye disease affects millions worldwide, and currently, there are limited options to slow its progression or reverse vision loss,” he predicted: “As more companies enter this space, the focus will shift towards therapies aimed at potentially restoring vision. With advancements in gene therapy, optogenetics, and other innovative approaches, there is a real opportunity to develop treatments that can not only slow disease progression but also provide hope for patients by reversing vision loss. As we move forward, I expect to see increased investment in these cutting-edge therapies, driven by the promise they hold for transforming the lives of those affected by RP in 2025.”
Women’s Health
“In 2025, we’ll see women’s health continuing to transition from a niche focus to a cornerstone of global healthcare,” said Kevin Ali, CEO of Organon. “Reproductive health remains essential and foundational, and we must reinforce its importance. We must also evolve the definition of women’s health to include conditions that disproportionately affect women, such as cardiovascular diseases, skin conditions and migraines. With this holistic approach, we can move closer to closing the innovation gap in these areas and redefine care for women.”
Colleen Acosta is CEO of Freya Biosciences, which is taking an immunotherapy approach to treating reproductive disorders. She said: “Groundbreaking science, particularly in the I&I [immunology and inflammation] space, continues to emerge with the potential to positively impact patients. This is particularly encouraging for companies focused on women’s health, as investors and healthcare systems increasingly recognize the broader value of advancing therapeutics in this critical area.”
We must evolve the definition of women’s health to include conditions that disproportionately affect women, such as cardiovascular diseases, skin conditions and migraines.
Kevin Ali, Organon
Genetic Medicines
“Gene therapy has the potential to change the way medicine is delivered for millions, and we are only at the beginning of seeing its impact,” said Curran Simpson, CEO of REGENXBIO. “I believe the next wave of gene therapy innovation will be even more transformative, including expanding gene therapies beyond rare disease communities. Clinical researchers are exploring gene therapies for common conditions that affect the masses, such as retinal, cardiovascular, neuromuscular, and metabolic diseases. As we continue to advance this science, identify additional gene delivery mechanisms, and invest in quality manufacturing and process development, significant breakthroughs are on the horizon. With researchers, physicians, sponsors, payers, and patient communities working together, we can fully realize the curative potential of gene therapy.”
Purespring Therapeutics’ CEO Julian Hanak, had a similar perspective. “As we look ahead to 2025, gene therapy is poised to expand beyond rare genetic disorders into more common conditions and new therapeutic areas,” he said, going on to note that “nephrology is one example of an area of medicine where we expect to see advances in gene therapy really impact the treatment of disease. With increased approvals and clinical trials anticipated in 2025, gene therapy will provide new hope and lasting for patients with previously difficult-to-treat conditions, marking a paradigm shift in the existing landscape.”
As we look ahead to 2025, gene therapy is poised to expand beyond rare genetic disorders into more common conditions and new therapeutic areas.
Julian Hanak, Purespring Therapeutics
Michael Parini, CEO of Spur Therapeutics, said: “Gene therapy is at an inflection point. We’ve learned a lot from the first wave of therapies – most importantly that the modality as the innovation isn’t enough. We can’t settle for a more convenient one-time therapy that doesn’t deliver better outcomes. What’s exciting is that science is evolving to allow us to unlock the full potential of gene therapy and create truly transformative medicines tailored to a disease. By optimizing every component of a gene therapy, selecting or designing the right capsid, optimizing the genome for the most efficient cellular interaction, and engineering the protein to have the right exposure and efficacy, we see huge potential for developing gene therapies that set new standards of care – not just for genetic diseases but also for more prevalent conditions where the consistent delivery of a therapeutic protein could change the course of disease for the people who need it most.”
For 64x Bio CEO Alexis Rovner, “It’s clear that more companies will need to find solutions to overcome bottlenecks in gene therapy manufacturing. The rapid advancements in gene editing and viral vector development are driving the need for scalable and efficient manufacturing processes. Current limitations in production capacity, quality control, and cost-effectiveness are hindering the widespread adoption of gene therapies.
“To address these challenges, companies will need to innovate in areas such as viral vector production, process automation, and supply chain management. By developing advanced manufacturing technologies, we can overcome these bottlenecks and bring more gene therapies to market,” Rovner said. “At the forefront of this effort, we’re seeing increased collaboration between biotech companies, contract manufacturing organizations, and technology providers. In 2025, I expect to see significant progress in gene therapy manufacturing, enabling the delivery of transformative treatments to patients more quickly and efficiently.”
In 2025, I expect to see significant progress in gene therapy manufacturing, enabling the delivery of transformative treatments to patients more quickly and efficiently.
Alexis Rovner, 64x Bio
“Expanding gene therapy to more common diseases is an exciting but complex challenge that requires overcoming scientific, regulatory, and logistical hurdles,” said Bruce Lahn, chief scientist of VectorBuilder. “While many rare diseases are monogenic, making them more straightforward targets for gene therapy, common diseases often involve multiple genes and pathways that may not yet be fully understood. This complexity necessitates innovative approaches to gene delivery and expression that can address polygenic influences and multi-pathway interactions.
“From a regulatory perspective, novel therapies for diseases with well-established treatments must demonstrate clear advantages in safety, efficacy, and accessibility,” Lahn added. “The larger patient populations associated with common diseases also present unique challenges. For example, rare adverse events, which may be negligible in smaller populations, could become more prominent and pose legal and ethical considerations for therapy developers. Rigorous clinical testing and long-term follow-up will be crucial in managing these risks.”
For Lahn, “technological advancements in delivery methods offer promising solutions to many of these challenges. While AAV remains a cornerstone for in vivo gene delivery, its limitations, such as targeting specificity and efficiency, highlight the need for continued innovation. Developing new delivery modalities, such as lipid nanoparticles (LNPs) for DNA or RNA, and engineered viral capsids with improved specificity, represents a significant step forward. Additionally, advancements in large-gene and multi-gene delivery technologies will enable researchers to address common diseases' more complex genetic architecture.
“The development of precision delivery systems tailored to specific disease contexts is proving to be a key enabler in expanding gene therapy’s potential. These systems enhance targeting accuracy, reduce off-target effects, and address the scalability needed for treating larger patient populations. As the field continues to innovate, these advancements in delivery technologies will play a central role in overcoming the scientific and logistical hurdles associated with applying gene therapy to common diseases, ultimately broadening its impact on global health.”
“Genetic medicines will grow to dominate the healthcare landscape due to [their] tremendous potential to transform medical treatment across multiple diseases. The combination of technological innovation, increased understanding of genetic mechanisms, and substantial investment suggests a promising future for genetic medicines next year and beyond,” said ViaNautis CEO Adi Hoess.
“At [their] core, genetic medicines rely on two fundamental attributes that enable their growth: the therapeutic cargo itself and the ability to deliver it to specific cell types, which will enable multi-market opportunities. The recent technological innovations in cell-specific delivery continue to push the boundaries of what’s possible, offering hope for more effective and precise therapies for a large of patients suffering from genetic diseases.”
“Epigenetics is an emerging frontier in medicine that saw significant growth in 2024, with increasing numbers of companies and investments driving progress in the field,” commented Jim Brown, CEO of DURECT.
“Epigenetic approaches offer immense potential to revolutionize therapies across various disease areas by leveraging the body’s natural mechanisms for gene regulation,” he said. “This approach enables the alteration of thousands of genes' expression simultaneously by targeting a single protein or small group of proteins such as DNMTs [DNA methyltransferases]. Recent advances in understanding epigenetic regulators have expanded the scope of epigenetics beyond its traditional applications in cancer. Companies are evaluating its potential in fields like tissue regeneration, including liver diseases, and other life-threatening conditions that are difficult to treat due to the complex interplay of proteins and signaling pathways.”
Epigenetic approaches offer immense potential to revolutionize therapies across various disease areas by leveraging the body’s natural mechanisms for gene regulation.
Jim Brown, DURECT
“While the study of genetics has provided an incredible insight into the factors driving disease, we still lack the tools and strategies to fully transform those insights into medicines, particularly when it comes to diseases due to under-expression of key proteins,” said Josh Mandel-Brehm, CEO of CAMP4. “While we have an arsenal of small molecules and biologics that employ diverse mechanisms of action to downregulate the expression of specific genes, there are no broadly applicable therapeutic strategies to date that enable us to turn up gene expression and restore healthy protein levels. This gap has held back us from developing transformative treatments for a significant number of diseases characterized by reduced protein levels. Recent breakthroughs in our understanding of gene regulation, including the role played by regulatory elements controlling transcription, are filling this critical gap and paving the road for our industry to advance entirely new therapeutic strategies in 2025 and beyond.”
“Gene expression governs all aspects of cellular physiology and is implicated in nearly every disease. While strategies to downregulate or silence specific genes exist, durably upregulating gene expression has remained a significant challenge,” also pointed out Kaan Certel, CEO of Omega Therapeutics. “However, 2025 promises new approaches to potentially unlock this goal. Epigenetics, the natural mechanism regulating gene expression, offers a promising and elegant avenue for therapeutically relevant upregulation without altering DNA sequence. By leveraging advances in computational tools, we can prospectively identify highly specific genomic targets associated with disease and the necessary epigenetic modification to achieve precise and desired changes in expression. This approach has the potential to address a wide range of conditions, including metabolic disorders and tissue regeneration.”
“RNA editing is a powerful approach with significant therapeutic potential to treat genetic disorders with high unmet need, offering notable advantages over gene therapies,” said Daniel de Boer, CEO of ProQR. “This technology allows for precise targeting of specific RNA molecules without modifying the entire genome, thus minimizing off-target effects. Uniquely, RNA editing also offers the advantage of non-permanent changes, as the modified RNA quickly breaks down in the body, reducing the risk of permanent alterations and allowing therapies to be easily discontinued as needed. Companies like ProQR are leveraging RNA editing to treat genetic diseases in patients with limited treatment options, such as cholestatic diseases. We look forward to continuing to see substantial growth and progress in RNA editing in 2025, with expanded clinical programs and breakthroughs offering life-changing therapies to patients.”
“In 2025, I believe we’ll see progress and growing interest in RNAi technologies and the potential impact these therapies can have on rare diseases and complex conditions,” said Dee Datta, CEO of Switch Therapeutics. “The versatility of RNAi therapies can empower the investigation of treatments for complex conditions that are highly prevalent yet still difficult to treat, such as neurodegenerative diseases. The central nervous system is one area that may see increased innovation as a result of gene therapy, as researchers leverage it to unlock a level of precision that enables them to develop biomarkers for highly specific targets.”
“The award of the 2024 Nobel Prize in Medicine to the scientists who discovered microRNA will lead to the reassessment of microRNA as a therapeutic modality,” predicted Derek Gilchrist, CEO of Causeway Therapeutics. “Interest in the therapeutic potential of microRNA followed a classic ‘hype cycle,’ peaking in the early 2010s and falling in the second half of the decade. I predict that we are headed up the ‘slope of enlightenment,’ as companies learn to work with the limitations of microRNA as a therapeutic modality, in particular the challenges involved in delivery of the compound to its target tissue. I believe that new approaches to delivery will help the industry realize the promise of microRNAs as a pleiotropic therapeutic modality that can treat some diseases at their root cause.”
The award of the 2024 Nobel Prize in Medicine to the scientists who discovered microRNA will lead to the reassessment of microRNA as a therapeutic modality.
Derek Gilchrist, Causeway Therapeutics
Cell Therapy
Predictions around cell therapy included expectations of expansion in the number and type of diseases that can be addressed, and improvements to the technology to enable faster, cheaper, more scalable manufacturing.
“With recent regulatory approvals of two cell therapies for solid tumors, it is clear that a single dose of T-cells can deliver transformative efficacy in late-stage patients with difficult to treat cancers,” said Adrian Rawcliffe, CEO of Adaptimmune. “We can now see the potential to fundamentally change the treatment of cancer as additional T-cell therapies become available in the years to come.”
“There have been tremendous achievements in the field of cell therapy to treat solid tumor cancers, with some notable FDA approvals in 2024,” agreed Thomas Soloway, CEO, of T-knife Therapeutics. “However, durable responses have been limited outside of melanoma and synovial sarcoma. As a result, in 2025, there will be an increased focus on the engineering of additional T-cell enhancements to create third-generation cell therapies with improved fitness and persistence that can increase durability of responses and broaden activity into additional solid tumors.”
In 2025, there will be an increased focus on the engineering of additional T cell enhancements to create third-generation cell therapies with improved fitness and persistence that can increase durability of responses and broaden activity into additional solid tumors.
Thomas Soloway, T-knife Therapeutics
“In 2025, our industry has both the challenge and privilege to continue to accelerate the pace of innovation, fueled by our mission to help more patients,” said Lynelle Hoch, president, cell therapy organization, at Bristol Myers Squibb. “BMS embodies this spirit of ‘what more can we do?’ In my role, I have seen the transformative potential of cell therapy become a reality, providing patients with certain, refractory blood cancers deep and durable responses, while improving engineering and logistics to meet growing patient demand.
“Looking forward, the number of patients we hope to treat expands exponentially as we bring cell therapy into a new frontier: autoimmune disease. While early, recent data has shown that it has the potential to trigger an ‘immune reset’ for patients living with serious, refractory diseases like systemic lupus, providing hope for treatment-free remissions not widely thought possible this time last year.
“It’s this progress – new hope for more patients – that motivates me, making each new year a moment of anticipation and renewed enthusiasm,” Hoch concluded.
“The next wave of cell therapy innovation in 2025 is set to transform patient care, offering one-time curative therapies for diseases where there remains tremendous unmet need,” said Dan Kirby, chief commercial officer, Orca Bio. “Advances in technology are designed to enable next-generation therapies with lower toxicity profiles, improving survival rates and quality of life while expanding possibilities in fields like oncology and autoimmune diseases.
“Achieving this promise requires unprecedented collaboration across biopharma, payers, healthcare providers and regulators to address challenges like equitable pricing, scalable manufacturing and sustainable reimbursement models,” Kirby noted. “The convergence of innovation, policy and partnership will be key to ensuring these life-saving treatments reach all who need them. 2025 stands to be a milestone year in delivering on the potential of these therapies, driving meaningful progress in global patient outcomes.”
“Cell therapy has broad potential to be transformative in multiple areas of unmet need, demonstrated by clinical results from autologous cell therapies in hematologic malignancies and autoimmune diseases,” said Atara Biotherapeutics’ CEO Cokey Nguyen. “It is becoming increasingly clear that a biology-first approach with no genetic editing may offer a unique advantage versus autologous and other allogeneic CAR-T platforms. Moving into early 2025, we remain optimistic in the potential of tabelecleucel to be the first-ever FDA-approved allogeneic T-cell therapy in the US, in addition to significant momentum for multiple clinical milestones from our allogeneic CAR-T programs in oncology and autoimmune diseases. Promising results from autologous therapies in autoimmune indications set the stage for the possibility of an allogeneic approach to attain ideal scale and accessibility, in addition to investigating the omission of lymphodepletion, which may limit adoption for some patients.”
“The effectiveness of autologous therapies for the treatment of cancer has been shown again and again. However, the number of patients successfully treated with these therapies is generally lower than most people assume - with only 30,000-40,000 patients treated globally since the inception of CAR-T as a therapy modality,” said Brian Burke, chief commercial officer of Tozaro. “Two significant factors for this low figure are cost and logistics. As a result, allogeneic treatments have been heralded as a more cost-effective option, although progress towards routine use of allogeneic cell platforms has been slow. There is a possibility these therapies may be bypassed altogether by in vivo CAR-T treatments using viruses - with a record number of clinical trials already underway. One recent highlight in this space saw Interius Biotherapeutics announce the first use of durable in vivo CAR therapy in the clinic to treat B-cell malignancies using a lentiviral vector. It’s certainly an interesting race in this space!”
With cell and gene therapies being approved for earlier lines of treatment, scaling manufacturing operations has become imperative. Traditional methods can no longer keep pace. The industry must adopt digitalization and industrialization to drive efficiency and meet growing needs.
Dan Strange, Cellular Origins
“In 2024, cell therapy approvals reached record levels, with the eligible patient population increasing logarithmically. This shift has turned a theoretical challenge into an urgent reality: a critical gap now exists between manufacturing capacity and patient demand,” said Dan Strange, chief technology officer at Cellular Origins. “With cell and gene therapies being approved for earlier lines of treatment, scaling manufacturing operations has become imperative. Traditional methods can no longer keep pace. The industry must adopt digitalization and industrialization to drive efficiency and meet growing needs.
“In 2025, we will see substantial investment in innovative manufacturing technologies from therapy developers and contract manufacturers,” Strange added. “We also expect to see more collaborative technology partnerships to provide a holistic manufacturing solution. These advancements are essential to fulfil the potential of cell therapy and ensure access for every eligible patient.”
Becky Cap, senior vice-president, Biotherapies, at Vitalant, told Scrip: “In 2025, the biopharma industry is expected to see significant advancements, with FasT CAR-T therapies emerging as a game changer [Editor’s note: Gracell developed the FasTCAR next-day autologous CAR-T cell manufacturing platform as outlined in an article in Nature]. These therapies promise quicker, more efficient production and delivery of CAR-T cells, potentially treating a broader range of cancers. The focus will be on optimizing manufacturing processes, expanding treatment settings, and improving patient access. This innovation could revolutionize cancer treatment, making it more accessible and effective.”
Transplantation And Regeneration
“In 2025, I believe we’ll see a breakthrough year for xenotransplantation, driven by significant advancements in genetic engineering and immunology,” predicted eGenesis CEO Mike Curtis. “At eGenesis, we’re pushing the boundaries of these technologies to make xenotransplantation a viable and scalable solution for addressing the organ shortage crisis. With improved gene editing techniques, such as CRISPR, we’re able to precisely modify donor pigs’ genomes to reduce immune rejection risks and enhance compatibility with humans for organ transplantation.
“Next year, we expect to see continued progress with our human-compatible kidney program and additional transplants. As we enter 2025, the combination of cutting-edge genetic technologies and enhanced clinical trial designs will bring xenotransplantation closer to the first FDA-approved clinical trial, offering hope to millions waiting for an organ transplant.”
For Elias Papatheodorou, CEO of HepaRegeniX, “a major frontier in human health is regeneration.” He said: “As our population ages, the need for regenerative solutions is greater than ever. I believe we’ll begin to see significant clinical progress in this field over the next year. Notably, many current approaches rely on complex technologies like cell therapies, tissue engineering, or gene therapies, which can limit patient access. To truly unlock regeneration’s potential, more accessible and scalable solutions are essential. We see small molecules as a promising path forward, and our developments in liver regeneration suggest their viability as a first step in this evolving field.”
As we enter 2025, the combination of cutting-edge genetic technologies and enhanced clinical trial designs will bring xenotransplantation closer to the first FDA approved clinical trial.
Mike Curtis, eGenesis
Therapeutic Antibodies
“Since their emergence as engineered treatment modalities, antibodies have evolved from single-target therapeutics into multi-functional tools, serving as standalone treatments and essential components of combination therapies across diverse diseases,” said Jeng Her, CEO of AP Biosciences. “These next-generation antibodies engage multiple pathways with precision, enabling conditional activation and multi-targeting, while offering greater therapeutic versatility and compatibility with other modalities.
“Antibodies have already transformed healthcare since the OKT3 approval in 1986, accounting for 140+ FDA approvals and generating more than $200bn in annual revenue globally, with applications in cancer, autoimmune diseases, and beyond,” Her added.
“Advances in bispecific antibodies exemplify this ‘Antibody 2.0’ era, enabling breakthroughs in previously untreatable diseases by engaging immune cells while targeting disease-specific markers. These therapies improve efficacy, reduce toxicity, and offer patient-friendly treatment options. As these innovations accelerate, 2025 will solidify bispecific antibodies as a backbone of modern medicine, driving new paradigms in precision and combination therapies.”
2025 will solidify bispecific antibodies as a backbone of modern medicine, driving new paradigms in precision and combination therapies.
Jeng Her, AP Biosciences
Catherine Bladen, vice president, regional executive and principal advisor at Vector Laboratories, said: “In 2025, recombinant technology will solidify its position as the gold standard in therapeutic antibody development. The advantages of recombinant antibodies – such as reproducibility, consistency and rapid customization – make them highly adaptable to evolving therapeutic needs. With the ability to precisely engineer properties like humanization and isotype switching within days, recombinant antibodies allow researchers to quickly tailor therapies for better efficacy and fewer side effects. As demand for personalized treatments and complex modalities like bispecifics grows, the flexibility of recombinant technology will be crucial in advancing new antibody-based therapies.”
Other Technology Advances
From innovations in biomarkers and target identification through drug delivery and manufacturing to digital health and techbio, our crystal-ball gazers shared many reasons to be excited about technology-driven progress in healthcare.
“Although significant breakthroughs have been made in areas such as cancer, obesity and autoimmune diseases, the pace of developing novel, first-in-class medicines has been limited,” noted John Lepore, CEO of ProFound Therapeutics and CEO-partner of Flagship Pioneering. “The pharma and biotech industries have largely focused on iterating therapeutic approaches to established drug targets (eg, PD-1, GLP-1).”
In Lepore’s view, “While the potential rewards for discovering and drugging new targets are immense for both patients and companies, the pursuit has been hindered by a scarcity of novel, tractable drug targets. Additionally, in today’s capital-constrained financial environment, leadership teams and investors often prioritize de-risked, later-stage assets.”
He continued: “However, emerging insights into the human genome and its regulation are unlocking significant opportunities for novel drug targets. Specifically, a growing area of scientific exploration involves the discovery and characterization of an entirely new class of proteins derived from previously uncharted regions of the genome – the expanded or ‘dark’ genome. Understanding these proteins as potential drugs and drug targets could usher in an exciting wave of first-in-class therapies, addressing unmet needs in oncology, immune and metabolic diseases, and beyond.”
A growing area of scientific exploration involves the discovery and characterization of an entirely new class of proteins derived from previously uncharted regions of the genome – the expanded or ‘dark’ genome. Understanding these proteins as potential drugs and drug targets could usher in an exciting wave of first-in-class therapies.
John Lepore, ProFound Therapeutics, Flagship Pioneering
“Harnessing the body’s natural protein degradation system to treat diseases has been a promising treatment modality that has gradually advanced in research and the clinic for nearly a decade. We’ve reached an exciting inflection point for the field of targeted protein degradation (TPD), with important clinical updates expected throughout the year from several small molecule degraders that have the potential to deliver transformative treatments for patients,” said Andrew Hirsch, CEO of C4 Therapeutics.
“Much like other advances in treatment modalities, such as antibodies and covalent inhibitors, TPD unlocks a vast potential to address multiple promising therapeutic targets, including those in oncology and autoimmune disorders. It is exciting to see a treatment method that has been in the making for so long emerge with such promise to make a real impact for patients.”
“Antibody-drug conjugates have taken off in the past couple of years, particularly in cancer treatment, and we expect the global market to continue growing in 2025 and beyond as we develop the new generation of ADCs and harness the full potential of the technology in comparison to what is currently offered to patients,” said Dragan Grabulovski, co-founder and acting CEO of Araris. “We are moving towards safer and more effective ADCs with improved payload and linker technologies that will not only make the treatment paradigm better for the patient, but also broaden the applications for ADCs across different therapeutic areas beyond cancer – expanding the possibilities beyond what was thought previously possible.”
“Clinically proven therapeutic modalities face high competition as exemplified by the explosion of antibody-drug conjugates in oncology or AAV-vectors in gene therapy. This is good for patients but delivers only incremental gains for market players. Thus, major improvements in specific therapeutic areas are likely to come from cross-fertilization of technologies and therapeutic modalities previously confined to selected fields,” said Victor Levitsky, chief scientific officer of Circio.
“One illustration of this is the ongoing transit of CAR-Ts from an oncology-focused approach into autoimmunity. Furthermore, the CAR-T technology per se will be requiring adjustments to comply with differences in tolerable toxicity and acceptable long-term complications in these two areas of medicine. To this end, RNA technologies, and especially circular RNA-based approach developed by Circio, may cross over to CAR-Ts from other fields which they were previously assigned to such as vaccination or gene therapy. It seems likely that near future will present us with more examples of such transitions,” Levitsky predicted.
“The nucleic acid delivery landscape in 2025 holds immense promise, particularly with anticipated breakthroughs in immuno-oncology. These advancements offer significant hope for cancer patients,” said Bernard Sagaert, CEO of etherna. “In other areas the transition from ex vivo autologous treatments to in vivo allogeneic approaches based on nucleic acid delivery can become game changing, such as for CAR-T therapies, opening therapeutic potential in oncology but also in the auto-immune space.
“A paradigm shift is also underway as the industry evolves from focusing solely on delivery mechanisms to embracing a holistic strategy. This integrated approach combines advancements in payload design, delivery optimization, and material quality, opening the door to transformative therapeutic possibilities,” Sagaert concluded.
“Therapeutic innovation is accelerating, with remarkable progress in oncology, neuroscience and gene therapies. In oncology, immunotherapies like CAR-T and personalized cancer vaccines are pushing boundaries, offering curative potential for previously untreatable cancers. Neuroscience is witnessing breakthroughs in Alzheimer’s and Parkinson’s disease, thanks to advancements in biomarkers, devices and novel drug mechanisms. New delivery technology, including hydrogel encapsulation, holds promise to improve both safety and efficacy while also reducing some of the complex logistics that drive up timelines and costs,” said Stella Vnook, CEO of Likarda.
“Gene and cell therapies are expanding rapidly, targeting rare genetic disorders with unprecedented precision. mRNA technologies, proven during the pandemic, are being adapted for vaccines and therapies beyond infectious diseases. Radiopharmaceuticals, which combine radioactive elements with targeting agents, are emerging as a promising modality in cancer treatment. This surge of innovation, driven by unmet medical needs, is reshaping the therapeutic landscape and offering hope to millions of patients,” Vnook expanded. “New delivery technology, including hydrogel encapsulation, holds promise to improve both safety and efficacy while also reducing some of the complex logistics that drive up timelines and costs.”
“Over the past decade, the biologics market has expanded nearly tenfold, with another tenfold increase anticipated in the next. This rapid growth has brought the industry to a critical crossroads: IV infusion resources are insufficient to support the influx of new therapies, and patients are demanding more convenient treatment options,” said Bryan Mazlish, CEO of Surf Bio. “To meet this challenge, one of the industry’s most urgent advancements will be the adoption of delivery technologies that enable self-administered, high-dose subcutaneous therapies. Companies with biologics on the market or in development must embrace a robust subcutaneous strategy to stay competitive – or risk falling behind. As biologics continue to proliferate, advancing subcutaneous delivery methods is essential to ensuring access to transformative therapies for patients worldwide.”
“Having supported the tech revolution in other sectors through our Level39 hub, at Canary Wharf Group we are really excited by the potential of techbio,” said Mairi Dillon, associate director, Science Ecosystem, at London specialist property group Canary Wharf Group. “We are seeing more early-stage companies emerge in this area with disruptive potential and the ability to identify new targets more efficiently. The life sciences industry has, understandably, traditionally been very conservative so the increasing sophistication with which companies are incorporating tech into their research and development is really exciting. Besides this, I am looking forward to seeing the latest developments in the fields of neuroscience and cell and gene therapy.”
Companies with biologics on the market or in development must embrace a robust subcutaneous strategy to stay competitive – or risk falling behind.
Bryan Mazlish, Surf Bio
David Benshoof Klein, CEO of Click Therapeutics, predicted: “The digitization of pharma pipelines is imminent. Since the FDA released its draft guidance on Prescription Drug Use-Related Software (PDURS) in 2023, which allows for any added clinically meaningful benefit from the use of software together with a drug to be added directly to the drug label, we’ve seen incredible interest from companies looking to add software-enhanced drug therapies to their portfolios. In 2025, we will start to see the development of smart ‘SE’ formulations of drugs, which combine a highly validated digital therapeutic with traditional pharmacotherapy to offer patients unique benefits including increased drug efficacy, optimized dosing, agile side effect management, and enhanced tolerability and persistence, ultimately improving treatment outcomes. These revolutionary treatments will leverage the latest technology to respond and adapt to patients to create personalized digital treatment journeys that can help bridge gaps in treatment and integrate seamlessly into pharmaceutical care.”
“While we expect new molecular entity discovery and development to continue in 2025, we also anticipate an increased focus on novel supportive innovations that will better meet the needs of patients living with complex and chronic diseases. This is especially true in plasma-derived therapies, where often patients receive these treatments for life,” commented Giles Platford, president of Takeda’s plasma-derived therapies (PDT) business unit.
“We are excited about the technology advances and formulation improvements that will allow us to optimize the delivery of current therapies and enhance the patient experience, while minimizing treatment burden. We also expect to see a strong surge of digital solutions, such as apps and connected devices, that will play an increasingly crucial role in helping to improve diagnostic accuracy and empowering patients to manage their therapies more effectively.”
We expect to see a strong surge of digital solutions, such as apps and connected devices, that will play an increasingly crucial role in helping to improve diagnostic accuracy and empowering patients to manage their therapies more effectively.
Giles Platford, Takeda
“These are exciting times for our industry, with more new therapies making it to market each year to transform patient lives. At the same time, many therapeutic developers continue to struggle with the spiralling cost to develop a drug candidate through to market,” observed Tomasz Kostrzewski, chief scientific officer of CN Bio. “Around 90% of drugs that make it into the clinic still ultimately fail, and addressing this hurdle is a key focus for the industry. Many of these later stage failures arise due to a lack of human-relevant data during preclinical development, but how do we solve this?”
Kostrzewski predicted a rise in tools to ensure clinical plans are informed by more accurate preclinical data: “New approach methodologies (NAMs) are already helping to revolutionize how we approach drug discovery, and will be a crucial growth area moving into 2025. Developed as viable, cost-saving and oftentimes more effective alternatives to existing methods, technologies such as organ-on-a-chip are helping therapeutic developers to progress their candidates to the clinic with more confidence than ever before. As we look towards the year ahead, we’re excited to see NAMs continue to go from strength to strength, enabling rapid and efficient therapeutic advancements.”
“Greater speed and efficiency will be paramount in 2025 as the life sciences community continues its push to develop new therapies and modalities faster. Partnerships will be key to this work. Companies will need collaborators that enable their ideas with solutions that deliver results with ease,” said Joe Fox, president of Beckman Coulter Life Sciences. “Many labs will require application expertise as they automate workflows to balance growing demands on their team. Others will seek new insights from small particles, like extracellular vesicles, as exciting new technologies revolutionize this workflow. The company that offers flexibility, scalability, and sustainability will emerge as the preferred partner.”
Diagnostics
“Advances in diagnostics are poised to revolutionize therapeutic development and delivery,” said Erez Podoly, vice president of R&D, Cleveland Diagnostics. “Spatial biology techniques, such as spatial transcriptomics and advanced immunohistochemistry, are uncovering cellular interactions within tissue environments, revealing disease mechanisms at unprecedented depth. These insights are advancing our understanding of cancer, neurodegenerative disorders, and cardiovascular diseases, enabling the identification of early biomarkers and novel therapeutic targets.”
Podoly said: “As healthcare shifts toward personalized medicine, diagnostics will play a pivotal role in tailoring therapies to individual patients. From early detection and risk stratification to guiding targeted treatments, diagnostics are transforming the therapeutic landscape, ensuring earlier interventions and more precise, effective care.”
As healthcare shifts toward personalized medicine, diagnostics will play a pivotal role in tailoring therapies to individual patients. From early detection and risk stratification to guiding targeted treatments, diagnostics are transforming the therapeutic landscape.
Erez Podoly, Cleveland Diagnostics
“Transformative companion diagnostics will be used increasingly to optimize cancer treatment by ensuring the right medicines are prescribed for the right patients,” said Karan Arora, senior vice president of advanced assays, AI, and pharma services at Leica Biosystems. “For example, tests to identify breast cancer patients who would benefit from antibody-drug conjugates will maximise the potential of these next-generation targeted therapies.”
“The biotechnology industry is poised to make a major impact in 2025 as it works to address the rising demand for in vitro diagnostics (IVDs) tailored for post-acute infection syndromes (PAIS), such as Long COVID, ME/CFS [myalgic encephalomyelitis/chronic fatigue syndrome], and PTLDS [post-treatment Lyme disease syndrome],” said James Foster, CEO of Virax Biolabs. “These chronic, complex conditions affect hundreds of thousands of patients each year, creating a significant healthcare burden due to lengthy and costly diagnostic processes at present. Emerging in vitro diagnostics specifically designed for PAIS could streamline diagnosis, making it faster, more accurate, and less costly. As these innovations advance, biotech companies have the opportunity to transform PAIS care by pushing these IVDs to market, facilitating the improvement of patient outcomes and reduction of strain on healthcare resources.”
Accessibility
Therapeutic advances are only as useful if patients can access them.
“The healthcare industry in 2025 will see a strong focus on improving access to care in underserved regions, focusing on therapeutic advancements to address the significant barriers to accessing essential services in the US,” said David Jones, senior medical director, neuroimmunology, at Genentech. “We’re witnessing significant developments with the rise of telehealth and innovative drug delivery methods, particularly for chronic conditions like multiple sclerosis. The approval of subcutaneous formulations, which address the potential access challenges associated with traditional IV therapies, is a testament to this progress. But there is still work to be done. This is an exciting time for expanding access to underserved populations, with new standards being set in drug R&D with these new therapeutic options.”
Jon Martin, head of Organon’s US biosimilar business unit, pointed out that “in 2025, we can anticipate costs will remain one of the biggest challenges in healthcare in the US. Biosimilars present a unique solution, and greater biosimilar adoption may help improve affordability by driving competition, reduce costs for the healthcare system and, above all else, potentially help increase access to patients.”
“Recent advancements in therapeutic areas such as oncology, gene therapy, and digital health have not only transformed healthcare outcomes but also underscored the need for accessibility and affordability,” said Novo Holdings Asia partner Navjeewan Khosla. “Asia, home to 60% of the world’s population, is a critical focus for these innovations. For example, India’s pharmaceutical industry is leveraging biosimilars to provide cost-effective alternatives to biologic therapies for oncology and autoimmune diseases. Similarly, China’s growing biotech sector has made strides in CAR-T therapies, with local companies delivering cutting-edge treatments at a fraction of global costs. Digital health platforms are also bridging gaps in accessibility, reducing barriers to care in underserved areas.
“From an investor perspective, these developments present a dual opportunity: driving strong returns while addressing critical social needs. The key lies in supporting scalable, innovative solutions that align with regional healthcare policies and infrastructure,” Khosla continued. “As healthcare demands continue to rise, fostering inclusive innovation will not only transform lives but also redefine value creation in the sector.”
As healthcare demands continue to rise, fostering inclusive innovation will not only transform lives but also redefine value creation in the sector.
Navjeewan Khosla, Novo Holdings Asia
